About the Authors

Francesco Muntoni

Roles Conceptualization, Investigation, Validation, Visualization, Writing – original draft, Writing – review & editing

f.muntoni@ucl.ac.uk

Affiliation NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, University College London, & Great Ormond Street Hospital Trust, London, United Kingdom

Michela Guglieri

Roles Conceptualization, Investigation, Validation, Visualization, Writing – original draft, Writing – review & editing

Affiliation The John Walton Muscular Dystrophy Research Centre, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, United Kingdom

Jean K. Mah

Roles Conceptualization, Investigation, Validation, Visualization, Writing – original draft, Writing – review & editing

Affiliation Cumming School of Medicine, University of Calgary, Alberta Children’s Hospital, Calgary, Alberta, Canada

Kathryn R. Wagner

Roles Conceptualization, Investigation, Validation, Visualization, Writing – original draft, Writing – review & editing

Affiliation Center for Genetic Muscle Disorders, and Departments of Neurology and Neuroscience, Kennedy Krieger Institute, Johns Hopkins School of Medicine, Baltimore, Maryland, United States of America

John F. Brandsema

Roles Conceptualization, Investigation, Validation, Visualization, Writing – original draft, Writing – review & editing

Affiliation Division of Neurology, The Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, United States of America

Russell J. Butterfield

Roles Conceptualization, Investigation, Validation, Visualization, Writing – original draft, Writing – review & editing

Affiliation University of Utah School of Medicine, Salt Lake City, Utah, United States of America

Craig M. McDonald

Roles Conceptualization, Investigation, Validation, Visualization, Writing – original draft, Writing – review & editing

Affiliation Lawson Health Research Institute, Children’s Hospital, London, Ontario, Canada

Anna G. Mayhew

Roles Conceptualization, Investigation, Validation, Visualization, Writing – original draft, Writing – review & editing

Affiliation Institute of Genetic Medicine, Newcastle University, Newcastle, United Kingdom

Jeffrey P. Palmer

Roles Conceptualization, Data curation, Formal analysis, Investigation, Methodology, Software, Supervision, Validation, Visualization, Writing – original draft, Writing – review & editing

Affiliation Pfizer Inc, Cambridge, Massachusetts, United States of America

Shannon Marraffino

Roles Conceptualization, Data curation, Funding acquisition, Investigation, Methodology, Project administration, Resources, Supervision, Validation, Visualization, Writing – original draft, Writing – review & editing

Affiliation Pfizer Inc, Cambridge, Massachusetts, United States of America

Lawrence Charnas

Roles Conceptualization, Data curation, Funding acquisition, Investigation, Methodology, Project administration, Resources, Software, Supervision, Validation, Visualization, Writing – original draft, Writing – review & editing

Affiliation Pfizer Inc, Cambridge, Massachusetts, United States of America

Eugenio Mercuri

Roles Conceptualization, Investigation, Validation, Visualization, Writing – original draft, Writing – review & editing

Affiliation Paediatric Neurology, Catholic University, and Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy

Competing Interests

F Muntoni has received support from, and/or has served as a paid consultant for, Avexis, Biogen, Dyne Therapeutics, Roche, Novartis, Pfizer, PTC Therapeutics, Sarepta Therapeutics, Santhera, the National Institute of Health Research Biomedical Research Centre at Great Ormond Street Hospital for Children NHS Foundation Trust, and University College London. M Guglieri has received support from, and/or has served as a paid consultant for, PTC Therapeutic, Capricorn, Marathon Pharmaceutical, Pfizer, ReveraGen, Italfarmaco SpA, WAVE Life Sciences, Santhera, Roche, Sarepta and Dynacure; and grant funding from H2020 and NIH. J Mah has received support from Pfizer, Sarepta Therapeutics, Bristol-Myers Squibb, Roche, NS Pharma, ReveraGen Biopharma, Catabasis, Italfarmaco SpA, and PTC Pharmaceuticals. K Wagner has received support from, and/or has served as a paid consultant for, Dynacure, PTC Therapeutics, Roche, Asklepios Biopharmaceutical Inc, Pfizer, Sarepta Therapeutics, Catabasis, and Vita Therapeutics. K Wagner is currently an employee of F. Hoffmann-La Roche Ag. JF Brandsema has received support from, and/or has served as a paid consultant for, Alexion, Audentes, AveXis, Biogen, Genentech, PTC Therapeutics, Sarepta, WAVE Life Sciences, CSL Behring, Cytokinetics, Fibrogen, Pfizer, and Summit. RJ Butterfield has received support from, and/or has served as a paid consultant for, Avexis, Caspricor Therapudics, Catabasis, PTC Therapeutics, Sarepta Therapeutics, Pfizer, Biogen, Avexis, and Acceleron. CM McDonald has received support from, and/or has served as a paid consultant for, PTC Therapeutics, Santhera Pharmaceuticals, Catabasis Pharmaceuticals Inc, Sarepta Therapeutics, Prosensa, Biomarin Pharmaceutical, Pfizer, Eli Lilly, Halo Therapeutics, Marathon Pharmaceuticals, Bristol-Myers Squibb, Novartis, Italfarmaco, Astellas / Mitobridge, Epirium Bio (formerly Cardero Therapeutics), Edgewise Therapeutics, Avidity Biosciences, Entrada Therapeutics. AG Mayhew has received support from, and/or has served as a paid consultant for, PTC Therapeutics, Biogen, BMS, Roche, Sarepta, Santhera, Italfarmaco, Catabasis, Mallinckrodt, Pfizer, Summit, Wave Biogen, and Avexis. J Palmer, L Charnas, and S Marraffino are employees of and own stock in Pfizer. EM Mercuri has worked as a PI and on advisory boards for Pfizer, PTC Therapeutics, Roche, Sarepta Therapeutics, Santhera, and NS. This does not alter our adherence to PLOS ONE policies on sharing data and materials.