Document is current

Article History

Accepted: 28 November 2024

First Online: 6 January 2025

Competing interests

: BioMarin provided financial support for this project. In addition to the individual competing interests outlined below, all authors declare travel to the meeting and accommodation reimbursement, and honorarium payments for meeting participation and Delphi voting (on which this manuscript is based) from BioMarin Pharmaceutical Inc. R.S. reported receiving consulting fees and grants from BioMarin, participation on advisory boards with Ascendis and BioMarin, and consulting for BridgeBio. J.H.-F. reported grants or contracts for clinical trials from BioMarin, QED and Pfizer, consulting fees from BioMarin, QED, NovoNordisk and Innoskel, honoraria from Medscape and BioMarin, participation in Advisory Board meetings for MCDS-Therapy Clinical Trial (unpaid), BioMarin and QED. K. Ozono reported receiving honoraria from Alexion, Kyowa Kirin, and BioMarin. P.B. reported participating on a data monitoring committee and receiving consulting fees and honoraria from BioMarin. V.C.-D. reported payment or honoraria for lectures/presentations from BioMarin and Ipsen, and participation in Advisory Board meetings for BioMarin, QED-Propel and Mereo. K.DeA. reported participating on advisory boards for BioMarin and BridgeBio and attending meetings with BioMarin, BridgeBio, Ascendis, and Tyra Biosciences. P.I. reported honoraria and support for attending meetings from BioMarin and a start-up grant to her institution from BioMarin. M.I. reported honoraria for consultancy services from BioMarin, QED Therapeutics, Pfizer/Therachon, Sanofi, Ascendis, Alexionk, Kyowa Kirin, Innoskel and NovoNordisk. J.L.J. reported participation on a data safety monitoring board/advisory board, receiving consulting fees and honoraria from BioMarin and Sanofi. M. Maghnie reported participating on a data safety monitoring/advisory board and receiving consulting fees and honoraria from Pfizer, Novo Nordisk, Merck, Sandoz and BioMarin. N.M. reported receiving consulting fees from Pfizer, BioMarin and Alexion, and honoraria from Medscape, Springer, Science Collected and TouchIM. N.M. also reported receiving support for attending meetings from Parent Project of Muscular Dystrophy and PESTOLA and holds a leadership or fiduciary role in the Board of Positive Exposure and the ACMG DEI committee. K.M. reported grants or contracts for clinical trials, honoraria and support for attending meetings, and has participated in Advisory Board meetings for BioMarin. K. Okada reported payment or honoraria for lectures/presentations from BioMarin and Alexion, and participation in advisory board meetings for BioMarin. S.N.I., M. Menzel and S.O.F. declare no additional competing interests.

Free to read: This content has been made available to all.