1Department of Pediatric Neurology, Catholic University, Rome, Italy
2Centro Clinico Nemo, Fondazione Policlinico Agostino Gemelli IRCCS, Rome, Italy
3Neuromuscular Unit, Department of Neurology and Research in Neuromuscular Diseases, Institut de Recerca Sant Joan de Déu, Center for Biomedical Research Network on Rare Diseases (CIBERER), ISCIII, Barcelona, Spain
4UCL Great Ormond Street Institute of Child Health, London, UK
5National Institute for Health Research, Great Ormond Street Institute of Child Health Biomedical Research Centre, University College London, London, UK
6Parent Project APS, Rome, Italy
7Hôpital Necker-Enfants Malades, Paris, France
8Department of Neuropediatrics and Muscle Disorders, Medical Center-University of Freiburg, Faculty of Medicine, Freiburg, Germany
9Department of Pediatrics, Gothenburg University, Queen Silvia Children’s Hospital, Gothenburg, Sweden
10Department of Neurology, Faculty of Medicine, University of São Paulo, São Paulo, SP Brazil
11Therapeutic Research in Neuromuscular Disorders Solutions (TRiNDS), Pittsburgh, PA USA
12Center for Genetic Medicine, Children’s National Health System and the George Washington, Washington, DC USA
13PTC Therapeutics Inc., South Plainfield, NJ USA
14PTC Therapeutics Germany GmbH, Frankfurt, Germany
15University of California Davis School of Medicine, Davis, CA USA