Spinal muscular atrophy (SMA) is one of the most common genetic diseases and was, until recently, a leading genetic cause of infant mortality. Three disease-modifying treatments have dramatically changed the disease trajectories and outcome for severely affected infants (SMA type 1), especially when initiated in the presymptomatic phase. One of these treatments is the adeno-associated viral vector 9 (AAV9) based gene therapy onasemnogene abeparvovec (Zolgensma®), which is delivered systemically and has been approved by the European Medicine Agency for SMA patients with up to three copies of the SMN2 gene or with the clinical presentation of SMA type 1. While this broad indication provides flexibility in patient selection, it also raises concerns about the risk-benefit ratio for patients with limited or no evidence supporting treatment. In 2020, we convened a European neuromuscular expert working group to support the rational use of onasemnogene abeparvovec, employing a modified Delphi methodology. After three years, we have assembled a similar yet larger group of European experts who assessed the emerging evidence of onasemnogene abeparvovec's role in treating older and heavier SMA patients, integrating insights from recent clinical trials and real-world evidence. This effort resulted in 12 consensus statements, with strong consensus achieved on 9 and consensus on the remaining 3, reflecting the evolving role of onasemnogene abeparvovec in treating SMA.

1st Department of Pediatrics «Hippokratio» General Hospital Aristotle University Thessaloniki Greece

Department of Clinical and Molecular Genetics Medicine Genetics Group University Hospital Vall d'Hebron Barcelona Spain

Department of Clinical Neurosciences for Children and Unit for Congenital and Hereditary Neuromuscular Disorders Department of Neurology Oslo University Hospital Oslo Norway

Department of Neurology Medical University of Warsaw Poland

Department of Neurology UMC Utrecht Brain Center University Medical Center Utrecht Utrecht University the Netherlands

Department of Neuropediatrics and Muscle Disorders Medical Center University of Freiburg Faculty of Medicine Freiburg Germany

Department of Pediatric Neurology University Hospitals Leuven and Department of Development and Regeneration KU Leuven Leuven Belgium

Department of Women's and Children's Health Karolinska Institutet Stockholm Sweden; Department Center for Neuromusculoskeletal Restorative Medicine Hong Kong Science Park Shatin New Territories Hong Kong China

Dept of Pediatric Neurology Motol University Hospital Prague Czech Republic

Division of Neuropaediatrics Development and Rehabilitation Department of Paediatrics Inselspital Bern University Hospital Bern Switzerland

Dubowitz Neuromuscular Centre UCL Great Ormond Street Institute of Child Health and NIHR Biomedical Research Centre Great Ormond Street Hospital for Children London UK

Heidelberg University Medical Faculty Heidelberg Center for Pediatric and Adolescent Medicine Department 1 Division of Pediatric Neurology and Metabolic Medicine Germany

Neuromuscular Centre Department of Pediatrics and Adolescent Medicine Clinic Favoriten Vienna Austria

Neuromuscular Reference Center Department of Pediatrics University Hospital Liège and University of Liège Belgium; MDUK Oxford Neuromuscular Centre and NIHR Oxford Biomedical Research Centre University of Oxford Oxford UK

Neuromuscular Unit Child Neurology and ICU Department Raymond Poincaré University Hospital APHP Paris Saclay Garches France

Pediatric Neurology Clinic Prof Dr Al Obregia Hospital Bucharest Faculty of Medicine and Pharmacy Carol Davila Bucharest Romania

Pediatric Neurology Institute Dana Dwek Children's Hospital Tel Aviv Sourasky Medical Center Faculty of Medicine Tel Aviv University Israel

Pediatric Neurology Unit Hospital de Santa Maria Centro Hospitalar Universitário Lisboa Norte Lisbon Portugal

Pediatric Neurology Università Cattolica del Sacro Cuore and Centro Clinico Nemo Fondazione Policlinico Gemelli IRCCS Rome Italy

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