1. MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre, University of Oxford, Oxford, UK
2. Neuromuscular Reference Center, Department of Paediatrics, University and University Hospital of Liège, Liège, Belgium
3. Department of Neurology, Stanford University Medical Center, Stanford, CA, USA
4. Departments of Neurology and Pediatrics, Columbia University Irving Medical Center, New York, NY, USA
5. Department for Neuropediatrics and Muscle Disease, Medical Center – University of Freiburg, Faculty of Medicine, Freiburg, Germany
6. Department of Paediatric Neurology and Nemo Clinical Centre, Catholic University, Rome, Italy
7. The Dubowitz Neuromuscular Centre, University College London, Great Ormond Street Institute of Child Health & Great Ormond Street Hospital, London, UK
8. National Institute of Health Research, Great Ormond Street Hospital Biomedical Research Centre, London, UK
9. Children’s Hospital of The King’s Daughters, Norfolk, VA, USA
10. Department of Neurology, David Geffen School of Medicine at UCLA, University of California, Los Angeles, Los Angeles, CA, USA
11. Department of Clinical and Molecular Genetics, Hospital Vall d’Hebron, Barcelona, Spain
12. Garches Neuromuscular Reference Center, Child Neurology and ICU Department, APHP Raymond Poincare University Hospital (UVSQ Paris Saclay), Garches, France
13. Hôpital Necker Enfants Malades, APHP, Paris, France
14. Institute of Medical Genetics, Tokyo Women’s Medical University, Tokyo, Japan
15. Novartis Gene Therapies, Inc., Bannockburn, IL, USA
16. Institute for Precision and Individualized Therapy, Eshelman School of Pharmacy, University of North Carolina at Chapel Hill, Chapel Hill, IL, USA
17. Genomics, Biotech and Emerging Medical Technology Institute, National Association of Managed Care Physicians, Richmond, VA, USA
18. Novartis Gene Therapies Switzerland GmbH, Rotkreuz, Switzerland
19. Center for Outcomes Research, University of Massachusetts Medical School, Worcester, MA, USA
20. Center for Experimental Neurotherapeutics, St. Jude Children’s Research Hospital, Memphis, TN, USA