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Unrelated to the study, some of the authors report the following potential conflicts of interest: NA has participated in clinical trials with Amgen and Novartis. MER has received a study grant from Ascendis Biopharma, and consultancy fees from Ipsen Biopharmaceuticals and Ultragenyx, with funds to her institution. HJM has participated in clinical trials with Roche, PTC Therapeutics, ReveraGen, Catabasis, Novartis and Sarepta, been a consultant for Novartis Gene Therapies and received research support from Roche. UJD has been a consultant to, and participated in clinical trials with, ReveraGen Biopharma. LMW has been a consultant to, and participated in clinical trials with, Amgen, Novartis, PTC, ReveraGen, Catabasis, Ipsen, and Ultragenyx, with funds to her institution.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Conflict of interest"}}]}}