Sustained clinical benefit following systemic gene replacement therapy in Duchenne muscular dystrophy

HJM has been a consultant for Novartis Gene Therapies and Hoffman La Roche Limited. HL has provided consultancy and/or received financial support for research projectsand clinical trials from Amplo Biotechnology, AMO Pharma, Argenx, Biogen, Desitin, Fulcrum Therapeutics, Harmony Biosciences, KYE Pharmaceuticals, MiloBiotechnology, Novartis, Pfizer, PTC Therapeutics, Hoffman-La Roche Limited, Sanofi-Genzyme, Santhera, Sarepta, Satellos, Spark Therapeutics and Ultragenyx. HL receives support from the Canadian Institutes of Health Research (FoundationGrant FDN-167281), the Canadian Institutes of Health Research and Muscular Dystrophy Canada (Network Catalyst Grant for NMD4C), the Canada Foundation for Innovation (CFI-JELF 38412), and the Canada Research Chairs program (Canada Research Chair in Neuromuscular Genomics and Health, 950-232279). Not applicable for this type of publication.