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Article History

Accepted: 3 October 2025

First Online: 13 November 2025

Competing interests

: S.J.T. reports research grant funding over the past 3 years from the CHDI Foundation, the NIHR Clinical Research Network, UK Medical Research Council (MR/X008029/1), UK Dementia Research Institute (principally funded by the UK Medical Research Council), and the Wellcome Trust (223082/Z/21/Z). S.J.T. also reports that over the past 3 years consultancy fees for advisory services were paid to University College London Consultants, a wholly owned subsidiary of University College London from the following companies: Alchemab, Alnylam Pharmaceuticals, Annexon Bioscience, Arrowhead, Atalanta Therapeutics, Catapult, Design Therapeutics, EcoR1, F. Hoffman-La Roche, Harness Therapeutics, Ipsen, Iris Medicine, Latus Bio, LifeEdit, Novartis Pharma, Pfizer, Prilenia, Prime Global, PTC Therapeutics, Rgenta Therapeutics, SkyHawk, Takeda Pharmaceuticals, Triplet Therapeutics, UniQure Biopharma, Vertex Pharmaceuticals, Vico Therapeutics, and Wave Life Sciences. S.J.T. has also consulted for Abingworth, Andera Partners, Ascidian Therapeutics, EQT, Cure Ventures, FunctionRX, Harrisson & Star, IQVIA and LifeLink through the office of Celtic Phenomenon. In the past 3 years, in University College London Hospitals NHS Foundation Trust host clinical institution S.J.T. has received funding to run clinical trials for Alnylam Pharmaceuticals, F. Hoffman-La Roche, Novartis Pharma, PTC Therapeutics, and UniQure Biopharma. Within the past 36 months, D.G.M. has been a scientific consultant and/or received an honoraria or research contract from AMO Pharma, Dyne, F. Hoffman-La Roche, Function Rx, LoQus23, MOMA Therapeutics, Novartis, Ono Pharmaceuticals, Pfizer Pharmaceuticals, PTC Therapeutics, Rgenta Therapeutics, Sanofi, Sarepta Therapeutics Inc, Script Biosciences, Skyhawk Therapeutics, Triplet Therapeutics, and Vertex Pharmaceuticals. D.G.M. also had research contracts with AMO Pharma and Vertex Pharmaceuticals. D.G.M. is on the Scientific Advisory Board of the Myotonic Dystrophy Foundation and EuroDyMA (European Dystrophia Myotonica Association), is a scientific adviser to the Myotonic Dystrophy Support Group, and is a vice president for research of Muscular Dystrophy UK. G.B. is the founder and chief executive officer (part time) of Function Rx ltd. The authors declare that they have no other competing interests.