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M.S. has received consultancy honoraria from Ionis, Prevail, Orphazyme, Servier, Reata, GenOrph and AviadoBio, all unrelated to the present manuscript. M.A.E. is an employee of Pacific Biosciences. S.Z. received consultancy honoraria from Neurogene, Aeglea BioTherapeutics and Applied Therapeutics and is an unpaid officer of the TGP Foundation, all unrelated to the present manuscript. The other authors declare no competing interests.","order":1,"name":"Ethics","group":{"name":"EthicsHeading","label":"Competing interests"}}]}}