1 Department of Neurology, Columbia University Irving Medical Center, 180 Fort Washington Avenue # 552, New York, NY, 10032-3791, USA. cac3@cumc.columbia.edu.
2 Centre of Translational and Experimental Myology, IRCCS Istituto Giannina Gaslini, and Department of Neuroscience, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health-DINOGMI, University of Genoa, Genoa, Italy.
3 Department of Neurology, Stanford University, Palo Alto, CA, USA.
4 Division of Neuropediatrics, University Children's Hospital Basel, University of Basel, Basel, Switzerland.
5 Pediatric Neurology Institute, Catholic University and Nemo Pediatrico, Fondazione Policlinico Gemelli IRCCS, Rome, Italy.
6 Department of Neuropediatrics and Muscle Disorders, Faculty of Medicine, Medical Center-University of Freiburg, Freiburg, Germany.
7 Department of Neurology, Medical University of Warsaw, Warsaw, Poland.
8 ERN EURO-NMD, Warsaw, Poland.
9 Pharma Development, Safety, F. Hoffmann-La Roche Ltd, Basel, Switzerland.
10 PDMA Neuroscience and Rare Disease, F. Hoffmann-La Roche Ltd, Basel, Switzerland.
11 Roche Pharmaceutical Research and Early Development, Roche Innovation Center Basel, Basel, Switzerland.
12 Roche Products Ltd, Welwyn Garden City, UK.
13 Product Development Neuroscience, F. Hoffmann-La Roche Ltd, Basel, Switzerland.
14 The Dubowitz Neuromuscular Centre, NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College London, and Great Ormond Street Hospital Trust, London, UK.