Declaration of competing interest A.V. has received compensation for ad hocc advisory boards/consulting activity with Biogen, Novartis, AveXis, Sarepta Therapeutics, PTC Therapeutics, Scholar Rock, Fibrogen, AMO Pharma, Pfizer, Catalyst, Lupin, Entrada Therapeutics, and Italfarmaco outside of the submitted work. J.S. has received compensation for consulting activity with Pfizer, Sarepta, and Immunoforge outside of the submitted work. W.B.B. has received compensation for consulting activity for PTC Therapeutics, Sarepta Therapeutics, and Novartis, Inc. outside of the submitted work. S.M. has received compensation for advisory boards for Mirum Pharmaceuticals and Ipsen Global outside of the submitted work. M.W.B. has received compensation for ad hoc advisory boards Albireo (now Ipsen) and Mirum outside of the submitted work. Other authors report no disclosures.