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N.C.A. is on the Boards of Directors of Novartis, Charles River Laboratories and Maze Therapeutics, and the Scientific Advisory Board of Dyne Therapeutics. A.H.B. has received consulting fees from Astellas Gene Therapies, GLG Inc., Guidepoint Global, and F. Hoffman-La Roche, is on the Scientific Advisory Board of Kate Therapeutics, and holds equity in Kate Therapeutics and Kinea Bio. J.H.C. has acted as an investigator for studies with GW Pharma\/Jazz Pharmaceuticals, Zogenix\/UCB Pharma, Vitaflo, Stoke Therapeutics, and Ultragenyx. She has been a speaker and on advisory boards for Jazz Pharmaceuticals, UCB, Biocodex, and Nutricia; all remuneration has been paid to her department. J.C. is a member of the Drug Monitoring and Safety Board for Anavex Life Sciences Corp, the Endpoint Adjudication Committee for Taysha Gene Therapies, the Scientific and Medical Advisory Committees of the Childhood Dementia Initiative and the Rett Syndrome Association of Australia, and is the director of the Mito Foundation of Australia. S.W.S. served on the scientific advisory committee of Population Bio, Deep Genomics and intellectual property from his research held at the Hospital for Sick Children and licensed to Athena Diagnostics and Population Bio. S.W.S., R.D.C., and C.R.M. are Editors or Editorial Board Members of\n                      npj Genomic Medicine\n                      ; they were not part of the peer review or decision-making processes for this manuscript. S.M.W., A.McT., A.M.D., G.C., A.P., I.E.S., V.C., L.D.S., S.E.M.S., M.W., A.D., N.S., P.S., L.S.C., and K.N.N. report no competing interests.","order":1,"name":"Ethics","group":{"name":"EthicsHeading","label":"Competing interests"}}],"article-number":"13"}}