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B.B., G.T., C.H., D.M.S., D.E.F., P.K. and K.D.F. are former employees of Novartis Gene Therapies, Inc. W.K.C. received honoraria for participation in a scientific advisory board for Regeneron Genetics Center and received grant support from Biogen for a newborn screening study outside of this study. A.H.M.B. is a consultant for Novartis Gene Therapies, Inc., for this study and other work, and conducted research for Exicure, Inc., outside of this study. V.L.M., C.R.P., and M.C. have nothing to disclose. R.F.H. was a consultant for Cunningham Meyer & Vedrine, PC, and Phillips Parker Orbeson & Arnett, PLC, outside of this study. J.R.M. is a consultant for Novartis Gene Therapies, Inc., for this study and other work, and receives honoraria for participation in scientific advisory boards from Novartis Gene Therapies, Inc., for this study and other work. M.S. reports honoraria for participation in scientific advisory boards from Novartis Gene Therapies, Inc., for this study and other work, Biogen and Roche, and for scientific presentation in Biogen-sponsored symposia outside of this study. F.M. reports a grant from Biogen for newborn screening and for registries outside of this study. F.M. also reports honoraria for participation in scientific advisory boards from Novartis Gene Therapies, Inc., for this study and other work, Biogen and Roche, and for scientific presentation at their industry-sponsored symposia outside of this study. Novartis Gene Therapies, Inc., sponsored the STR1VE-US (NCT03306277) and STR1VE-EU studies (NCT03461289).","order":1,"name":"Ethics","group":{"name":"EthicsHeading","label":"Competing interests"}}]}}