Spinal muscular atrophy (SMA) used to be one of the most common genetic causes of infant mortality. New disease modifying treatments have changed the disease trajectories and most impressive results are seen if treatment is initiated in the presymptomatic phase of the disease. Very recently, the European Medicine Agency approved Onasemnogene abeparvovec (Zolgensma®) for the treatment of patients with SMA with up to three copies of the SMN2 gene or the clinical presentation of SMA type 1. While this broad indication provides new opportunities, it also triggers discussions on the appropriate selection of patients in the context of limited available evidence. To aid the rational use of Onasemnogene abeparvovec for the treatment of SMA, a group of European neuromuscular experts presents in this paper eleven consensus statements covering qualification, patient selection, safety considerations and long-term monitoring.

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

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 Metabolic Medicine Centre for Childhood and Adolescent Medicine University Hospital Heidelberg Germany

Department of Neuropediatrics University Hospital Bonn Bonn Germany

Department of Womeńs and Childreńs Health Karolinska Institutet Stockholm Sweden

Dept of Pediatric Neurology Motol University Hospital Prague Czech Republic

Dept of Pediatric Neurology University Hospitals Leuven Belgium

Dubowitz Neuromuscular Centre UCL Great Ormond Street Institute of Child Health and NIHR Biomedical Research Centre Great Ormond Street Hospital for Children London 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 Università Cattolica del Sacro Cuore Rome Italy; Centro Clinico Nemo Fondazione Policlinico Universitario Agostino Gemelli IRCCS Rome Italy

University of Liège Neuromuscular Reference Center Disease Department of Pediatrics Liege Belgium; MDUK Neuromuscular Center Department of Pediatrics University of Oxford UK

Eur J Paediatr Neurol. 2020 Sep;28:2-3. doi: 10.1016/j.ejpn.2020.08.005. PubMed

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Recombinant Adeno-Associated Virus Serotype 9 Gene Therapy in Spinal Muscular Atrophy