Declaration of competing interest O.A.H. has received compensation for ad hoc advisory boards with PTC therapeutics, Sarepta, Catalyst, and Percheron and speaker's bureau for Biogen and Genentech. S.E.M. has received compensation for ad hoc advisory boards/consulting for Sarepta, Novartis, and Atamyo Therapeutics. S.W. has received compensation for advisory boards for Sarepta. K.Y.B. has received compensation for consulting/advisory role with Biogen, UCB, Reata Pharmaceuticals, Pfizer, Catalyst Pharmaceuticals, and MyTomorrows. C.M.P. has received honoraria for advisory board participation from Biogen, Novartis, Roche, and Sarepta and speaker's fees from Biogen and Novartis. A.V. has received compensation for ad hoc advisory boards/consulting activity with Biogen, Novartis, AveXis, Sarepta, PTC, Scholar Rock, Fibrogen, AMO, Pfizer, Catalyst, Lupin, Entrada, MyTomorrows, Percheron, and Italfarmaco.