-
Je něco špatně v tomto záznamu ?
Efficacy and Safety of Vamorolone Over 48 Weeks in Boys With Duchenne Muscular Dystrophy: A Randomized Controlled Trial
UJ. Dang, JM. Damsker, M. Guglieri, PR. Clemens, SJ. Perlman, EC. Smith, I. Horrocks, RS. Finkel, JK. Mah, N. Deconinck, NM. Goemans, J. Haberlová, V. Straub, L. Mengle-Gaw, BD. Schwartz, A. Harper, PB. Shieh, L. De Waele, D. Castro, ML. Yang,...
Jazyk angličtina Země Spojené státy americké
Typ dokumentu randomizované kontrolované studie, časopisecké články
- MeSH
- biologické markery MeSH
- dítě MeSH
- Duchennova muskulární dystrofie * farmakoterapie MeSH
- lidé MeSH
- prednison škodlivé účinky MeSH
- předškolní dítě MeSH
- pregnadiendioly * škodlivé účinky MeSH
- Check Tag
- dítě MeSH
- lidé MeSH
- mužské pohlaví MeSH
- předškolní dítě MeSH
- Publikační typ
- časopisecké články MeSH
- randomizované kontrolované studie MeSH
BACKGROUND AND OBJECTIVES: Vamorolone is a dissociative agonist of the glucocorticoid receptor that has shown similar efficacy and reduced safety concerns in comparison with prednisone in Duchenne muscular dystrophy (DMD). This study was conducted to determine the efficacy and safety of vamorolone over 48 weeks and to study crossover participants (prednisone to vamorolone; placebo to vamorolone). METHODS: A randomized, double-blind, placebo-controlled and prednisone-controlled clinical trial of 2 doses of vamorolone was conducted in participants with DMD, in the ages from 4 years to younger than 7 years at baseline. The interventions were 2 mg/kg/d of vamorolone and 6 mg/kg/d of vamorolone for 48 weeks (period 1: 24 weeks + period 2: 24 weeks) and 0.75 mg/kg/d of prednisone and placebo for the first 24 weeks (before crossover). Efficacy was evaluated through gross motor outcomes and safety through adverse events, growth velocity, body mass index (BMI), and bone turnover biomarkers. This analysis focused on period 2. RESULTS: A total of 121 participants with DMD were randomized. Vamorolone at a dose of 6 mg/kg/d showed maintenance of improvement for all motor outcomes to week 48 (e.g., for primary outcome, time to stand from supine [TTSTAND] velocity, week 24 least squares mean [LSM] [SE] 0.052 [0.0130] rises/s vs week 48 LSM [SE] 0.0446 [0.0138]). After 48 weeks, vamorolone at a dose of 2 mg/kg/d showed similar improvements as 6 mg/kg/d for North Star Ambulatory Assessment (NSAA) (vamorolone 6 mg/kg/d-vamorolone 2 mg/kg/d LSM [SE] 0.49 [1.14]; 95% CI -1.80 to 2.78, p = 0.67), but less improvement for other motor outcomes. The placebo to vamorolone 6 mg/kg/d group showed rapid improvements after 20 weeks of treatment approaching benefit seen with 48-week 6 mg/kg/d of vamorolone treatment for TTSTAND, time to run/walk 10 m, and NSAA. There was significant improvement in linear growth after crossover in the prednisone to vamorolone 6 mg/kg/d group, and rapid reversal of prednisone-induced decline in bone turnover biomarkers in both crossover groups. There was an increase in BMI after 24 weeks of treatment that then stabilized for both vamorolone groups. DISCUSSION: Improvements of motor outcomes seen with 6 mg/kg/d of vamorolone at 24 weeks of treatment were maintained to 48 weeks of treatment. Vamorolone at a dose of 6 mg/kg/d showed better maintenance of effect compared with vamorolone at a dose of 2 mg/kg/d for most (3/5) motor outcomes. Bone morbidities of prednisone (stunting of growth and declines in serum bone biomarkers) were reversed when treatment transitioned to vamorolone. TRIAL REGISTRATION INFORMATION: ClinicalTrials.gov Identifier: NCT03439670. CLASSIFICATION OF EVIDENCE: This study provides Class I evidence that for boys with DMD, the efficacy of vamorolone at a dose of 6 mg/kg/d was maintained over 48 weeks.
2nd School of Medicine Charles University Prague Czech Republic
Alberta Children's Hospital Research Institute University of Calgary Canada
Alder Hey Children's NHS Foundation Trust Liverpool
Ann and Robert H Lurie Children's Hospital Chicago IL
BC Children's Hospital Research Institute Vancouver Canada
Binghamton University State University of New York Binghamton
Children's Hospital of Eastern Ontario Ottawa Ontario Canada
Children's Hospital of Richmond Richmond VA
Children's National Medical Center Washington DC
Department of Neurology Leiden University Medical Center
Department of Paediatric Neurology University Hospitals Leuven Belgium
Duke University School of Medicine Durham NC
From Carleton University Ottawa Ontario Canada
Hospital Quirónsalud Valencia Spain
Kids Neuroscience Centre The Children's Hospital at Westmead Australia
KU Leuven Department of Development and Regeneration
Leeds Teaching Hospitals Trust United Kingdom
Montreal Children's Hospital Quebec
Murdoch Children's Research Institute Melbourne Victoria Australia
Nemours Children's Hospital Orlando FL Dr Monduy is now with Nicklaus Children's Hospital Miami FL
Neuromuscular Centre Department of Pediatric Neurology Motol University Hospital
Neuromuscular Reference Center UZ Ghent
Neuropaediatrics Department Institut de Recerca Pediàtrica Hospital Sant Joan de Déu Barcelona Spain
P and A Kyriakou Children's Hospital Athens Greece
Queen Silvia Children's Hospital Gothenburg Sweden
Radboud University Medical Center Nijmegen the Netherlands
ReveraGen BioPharma Rockville MD
Royal Hospital for Children Glasgow United Kingdom
Santhera Pharmaceuticals Prattein Switzerland
Schneider Children's Medical Center Tel Aviv University Israel
UCLA Medical School Los Angeles CA
University of California Davis Sacramento
University of Colorado School of Medicine Children's Hospital Colorado Aurora
University of Ottawa Ontario Canada
University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center PA
Citace poskytuje Crossref.org
- 000
- 00000naa a2200000 a 4500
- 001
- bmc24006743
- 003
- CZ-PrNML
- 005
- 20240423155456.0
- 007
- ta
- 008
- 240412s2024 xxu f 000 0|eng||
- 009
- AR
- 024 7_
- $a 10.1212/WNL.0000000000208112 $2 doi
- 035 __
- $a (PubMed)38335499
- 040 __
- $a ABA008 $b cze $d ABA008 $e AACR2
- 041 0_
- $a eng
- 044 __
- $a xxu
- 100 1_
- $a Dang, Utkarsh J $u From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; "P&A Kyriakou" Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton
- 245 10
- $a Efficacy and Safety of Vamorolone Over 48 Weeks in Boys With Duchenne Muscular Dystrophy: A Randomized Controlled Trial / $c UJ. Dang, JM. Damsker, M. Guglieri, PR. Clemens, SJ. Perlman, EC. Smith, I. Horrocks, RS. Finkel, JK. Mah, N. Deconinck, NM. Goemans, J. Haberlová, V. Straub, L. Mengle-Gaw, BD. Schwartz, A. Harper, PB. Shieh, L. De Waele, D. Castro, ML. Yang, MM. Ryan, CM. McDonald, M. Tulinius, RI. Webster, HJ. Mcmillan, N. Kuntz, VK. Rao, G. Baranello, S. Spinty, AM. Childs, AM. Sbrocchi, KA. Selby, M. Monduy, Y. Nevo, JJ. Vilchez, A. Nascimento-Osorio, EH. Niks, IJM. De Groot, M. Katsalouli, JN. Van Den Anker, LM. Ward, M. Leinonen, AL. D'Alessandro, EP. Hoffman
- 520 9_
- $a BACKGROUND AND OBJECTIVES: Vamorolone is a dissociative agonist of the glucocorticoid receptor that has shown similar efficacy and reduced safety concerns in comparison with prednisone in Duchenne muscular dystrophy (DMD). This study was conducted to determine the efficacy and safety of vamorolone over 48 weeks and to study crossover participants (prednisone to vamorolone; placebo to vamorolone). METHODS: A randomized, double-blind, placebo-controlled and prednisone-controlled clinical trial of 2 doses of vamorolone was conducted in participants with DMD, in the ages from 4 years to younger than 7 years at baseline. The interventions were 2 mg/kg/d of vamorolone and 6 mg/kg/d of vamorolone for 48 weeks (period 1: 24 weeks + period 2: 24 weeks) and 0.75 mg/kg/d of prednisone and placebo for the first 24 weeks (before crossover). Efficacy was evaluated through gross motor outcomes and safety through adverse events, growth velocity, body mass index (BMI), and bone turnover biomarkers. This analysis focused on period 2. RESULTS: A total of 121 participants with DMD were randomized. Vamorolone at a dose of 6 mg/kg/d showed maintenance of improvement for all motor outcomes to week 48 (e.g., for primary outcome, time to stand from supine [TTSTAND] velocity, week 24 least squares mean [LSM] [SE] 0.052 [0.0130] rises/s vs week 48 LSM [SE] 0.0446 [0.0138]). After 48 weeks, vamorolone at a dose of 2 mg/kg/d showed similar improvements as 6 mg/kg/d for North Star Ambulatory Assessment (NSAA) (vamorolone 6 mg/kg/d-vamorolone 2 mg/kg/d LSM [SE] 0.49 [1.14]; 95% CI -1.80 to 2.78, p = 0.67), but less improvement for other motor outcomes. The placebo to vamorolone 6 mg/kg/d group showed rapid improvements after 20 weeks of treatment approaching benefit seen with 48-week 6 mg/kg/d of vamorolone treatment for TTSTAND, time to run/walk 10 m, and NSAA. There was significant improvement in linear growth after crossover in the prednisone to vamorolone 6 mg/kg/d group, and rapid reversal of prednisone-induced decline in bone turnover biomarkers in both crossover groups. There was an increase in BMI after 24 weeks of treatment that then stabilized for both vamorolone groups. DISCUSSION: Improvements of motor outcomes seen with 6 mg/kg/d of vamorolone at 24 weeks of treatment were maintained to 48 weeks of treatment. Vamorolone at a dose of 6 mg/kg/d showed better maintenance of effect compared with vamorolone at a dose of 2 mg/kg/d for most (3/5) motor outcomes. Bone morbidities of prednisone (stunting of growth and declines in serum bone biomarkers) were reversed when treatment transitioned to vamorolone. TRIAL REGISTRATION INFORMATION: ClinicalTrials.gov Identifier: NCT03439670. CLASSIFICATION OF EVIDENCE: This study provides Class I evidence that for boys with DMD, the efficacy of vamorolone at a dose of 6 mg/kg/d was maintained over 48 weeks.
- 650 _2
- $a lidé $7 D006801
- 650 _2
- $a mužské pohlaví $7 D008297
- 650 _2
- $a biologické markery $7 D015415
- 650 12
- $a Duchennova muskulární dystrofie $x farmakoterapie $7 D020388
- 650 _2
- $a prednison $x škodlivé účinky $7 D011241
- 650 12
- $a pregnadiendioly $x škodlivé účinky $7 D011244
- 650 _2
- $a předškolní dítě $7 D002675
- 650 _2
- $a dítě $7 D002648
- 655 _2
- $a randomizované kontrolované studie $7 D016449
- 655 _2
- $a časopisecké články $7 D016428
- 700 1_
- $a Damsker, Jesse M $u From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; "P&A Kyriakou" Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton $1 https://orcid.org/0009000506805875
- 700 1_
- $a Guglieri, Michela $u From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; "P&A Kyriakou" Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton $1 https://orcid.org/0000000284550637
- 700 1_
- $a Clemens, Paula R $u From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; "P&A Kyriakou" Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton
- 700 1_
- $a Perlman, Seth J $u From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; "P&A Kyriakou" Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton
- 700 1_
- $a Smith, Edward C $u From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; "P&A Kyriakou" Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton
- 700 1_
- $a Horrocks, Iain $u From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; "P&A Kyriakou" Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton
- 700 1_
- $a Finkel, Richard S $u From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; "P&A Kyriakou" Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton $1 https://orcid.org/0000000293517054
- 700 1_
- $a Mah, Jean K $u From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; "P&A Kyriakou" Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton $1 https://orcid.org/0000000257959955
- 700 1_
- $a Deconinck, Nicolas $u From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; "P&A Kyriakou" Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton
- 700 1_
- $a Goemans, Nathalie M $u From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; "P&A Kyriakou" Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton $1 https://orcid.org/0000000251577155
- 700 1_
- $a Haberlová, Jana $u From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; "P&A Kyriakou" Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton
- 700 1_
- $a Straub, Volker $u From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; "P&A Kyriakou" Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton $1 https://orcid.org/0000000190463540
- 700 1_
- $a Mengle-Gaw, Laurel $u From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; "P&A Kyriakou" Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton
- 700 1_
- $a Schwartz, Benjamin D $u From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; "P&A Kyriakou" Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton
- 700 1_
- $a Harper, Amy $u From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; "P&A Kyriakou" Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton
- 700 1_
- $a Shieh, Perry B $u From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; "P&A Kyriakou" Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton
- 700 1_
- $a De Waele, Liesbeth $u From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; "P&A Kyriakou" Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton
- 700 1_
- $a Castro, Diana $u From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; "P&A Kyriakou" Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton
- 700 1_
- $a Yang, Michele L $u From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; "P&A Kyriakou" Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton
- 700 1_
- $a Ryan, Monique M $u From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; "P&A Kyriakou" Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton $1 https://orcid.org/0000000163971910
- 700 1_
- $a McDonald, Craig M $u From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; "P&A Kyriakou" Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton $1 https://orcid.org/0000000287793220
- 700 1_
- $a Tulinius, Mar $u From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; "P&A Kyriakou" Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton
- 700 1_
- $a Webster, Richard I $u From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; "P&A Kyriakou" Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton
- 700 1_
- $a Mcmillan, Hugh J $u From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; "P&A Kyriakou" Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton $1 https://orcid.org/0000000189272018 $7 ntk20221164250
- 700 1_
- $a Kuntz, Nancy $u From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; "P&A Kyriakou" Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton
- 700 1_
- $a Rao, Vamshi K $u From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; "P&A Kyriakou" Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton
- 700 1_
- $a Baranello, Giovanni $u From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; "P&A Kyriakou" Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton
- 700 1_
- $a Spinty, Stefan $u From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; "P&A Kyriakou" Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton $1 https://orcid.org/000000015437820X
- 700 1_
- $a Childs, Anne-Marie $u From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; "P&A Kyriakou" Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton
- 700 1_
- $a Sbrocchi, Annie M $u From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; "P&A Kyriakou" Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton
- 700 1_
- $a Selby, Kathryn A $u From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; "P&A Kyriakou" Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton
- 700 1_
- $a Monduy, Migvis $u From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; "P&A Kyriakou" Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton
- 700 1_
- $a Nevo, Yoram $u From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; "P&A Kyriakou" Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton
- 700 1_
- $a Vilchez, Juan J $u From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; "P&A Kyriakou" Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton $1 https://orcid.org/0000000205322872
- 700 1_
- $a Nascimento-Osorio, Andres $u From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; "P&A Kyriakou" Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton
- 700 1_
- $a Niks, Erik H $u From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; "P&A Kyriakou" Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton $1 https://orcid.org/0000000158925143
- 700 1_
- $a De Groot, Imelda J M $u From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; "P&A Kyriakou" Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton
- 700 1_
- $a Katsalouli, Marina $u From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; "P&A Kyriakou" Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton
- 700 1_
- $a Van Den Anker, John N $u From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; "P&A Kyriakou" Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton
- 700 1_
- $a Ward, Leanne M $u From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; "P&A Kyriakou" Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton $1 https://orcid.org/0000000315579185
- 700 1_
- $a Leinonen, Mika $u From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; "P&A Kyriakou" Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton
- 700 1_
- $a D'Alessandro, Andrea L $u From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; "P&A Kyriakou" Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton
- 700 1_
- $a Hoffman, Eric P $u From Carleton University (U.J.D.), Ottawa, Ontario, Canada; ReveraGen BioPharma (J.M.D., J.N.V.D.A., E.P.H.), Rockville, MD; John Walton Muscular Dystrophy Research Centre (M.G., V.S.), Newcastle Hospitals NHS Foundation Trust and Newcastle University, United Kingdom; University of Pittsburgh School of Medicine and Department of Veterans Affairs Medical Center (P.R.C.), PA; University of Washington School of Medicine (S.J.P.), Seattle; Duke University School of Medicine (E.C.S.), Durham, NC; Royal Hospital for Children (I.H.), Glasgow, United Kingdom; Nemours Children's Hospital (R.S.F.), Orlando, FL. Dr. Finkel is now with St. Jude Children's Research Hospital, Memphis, TN; Alberta Children's Hospital Research Institute (J.K.M.), University of Calgary, Canada; Neuromuscular Reference Center (NMRC) (N.D.), UZ Ghent; KU Leuven Department of Development and Regeneration (N.M.G., L.D.W.); Department of Paediatric Neurology (N.M.G., L.D.W.), University Hospitals Leuven, Belgium; Neuromuscular Centre (J.H.), Department of Pediatric Neurology Motol University Hospital; 2nd School of Medicine Charles University in Prague (J.H.), Czech Republic; The Camden Group (L.M.-G., B.D.S.), St. Louis, MO; Children's Hospital of Richmond (A.H.), Richmond, VA; UCLA Medical School (P.B.S.), Los Angeles, CA; UT Southwestern Medical Center (D.C.), Dallas, TX; University of Colorado School of Medicine (M.L.Y.), Children's Hospital Colorado, Aurora; The Royal Children's Hospital (M.M.R.); Murdoch Children's Research Institute (M.M.R.), Melbourne, Victoria, Australia; University of California, Davis (C.M.M.), Sacramento; Queen Silvia Children's Hospital (M.T.), Gothenburg, Sweden; Kids Neuroscience Centre (R.I.W.), The Children's Hospital at Westmead, Australia; University of Ottawa (H.J.M.), Ontario, Canada; Ann & Robert H. Lurie Children's Hospital (N.K., V.K.R.), Chicago, IL; The Dubowitz Neuromuscular Centre (G.B.), National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College, London; Alder Hey Children's NHS Foundation Trust (S.S.), Liverpool; Leeds Teaching Hospitals Trust (A.-M.C.), United Kingdom; Montreal Children's Hospital (A.M.S.), Quebec; BC Children's Hospital Research Institute (K.A.S.), Vancouver, Canada; Nemours Children's Hospital (M.M.), Orlando, FL. Dr. Monduy is now with Nicklaus Children's Hospital, Miami, FL; Schneider Children's Medical Center (Y.N.), Tel Aviv University, Israel; Hospital Quirónsalud Valencia (J.J.V.), Spain; Neuropaediatrics Department (A.N.-O.), Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain; Department of Neurology (E.H.N.), Leiden University Medical Center; Radboud University Medical Center (I.J.M.D.G.), Nijmegen, the Netherlands; "P&A Kyriakou" Children's Hospital (M.K.), Athens, Greece; Children's National Medical Center (J.N.V.D.A.), Washington, DC; Children's Hospital of Eastern Ontario (CHEO) Research Institute (L.M.W.), Ottawa, Ontario, Canada; Santhera Pharmaceuticals (M.L.), Prattein, Switzerland; TRiNDS (A.L.D.A.), Pittsburgh, PA; and Binghamton University-State University of New York (E.P.H.), Binghamton $1 https://orcid.org/0000000164705139
- 773 0_
- $w MED00003491 $t Neurology $x 1526-632X $g Roč. 102, č. 5 (2024), s. e208112
- 856 41
- $u https://pubmed.ncbi.nlm.nih.gov/38335499 $y Pubmed
- 910 __
- $a ABA008 $b sig $c sign $y - $z 0
- 990 __
- $a 20240412 $b ABA008
- 991 __
- $a 20240423155453 $b ABA008
- 999 __
- $a ok $b bmc $g 2080999 $s 1216510
- BAS __
- $a 3
- BAS __
- $a PreBMC-MEDLINE
- BMC __
- $a 2024 $b 102 $c 5 $d e208112 $e 20240209 $i 1526-632X $m Neurology $n Neurology $x MED00003491
- LZP __
- $a Pubmed-20240412