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Article History

Received: 20 February 2026

Revised: 25 March 2026

Accepted: 31 March 2026

First Online: 4 May 2026

Declarations

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: The authors have no competing interests to declare that are relevant to the content of this article Outside of this context, JYH received honoraria for lecturing, consulting, and travel expenses for attending meetings from Biogen, Pfizer, Roche, LFB, Italfarmaco, Lupin and Novartis. AG has received consulting fees for Ipsen, Sanofi and Theravia. MM has received support for attending meetings and/or travel from CSL-Behring and Roche and has served on advisory boards for Johnson&Johnson and Roche. PL has received research grants, consulting fees, speaker’s honoraria, support for attending meetings and/or travel from Biogen, Roche, Alexion, Johnson&Johnson, Ad Scientiam, Amicus, Astellas, Sanofi, Shionogi. He has served on advisory boards for Biogen, Roche, Alexion, Johnson&Johnson, Ad Scientiam, Amicus, Astellas, Sanofi, Shionogi and declares no stocks or shares. The other authors report no relevant disclosures.

: The protocols for the French GSD-III registry, Accelerix and NatHis DM1 studies were approved by the local Ethics Committee (CPP–Ile de France VI–Groupe Hospitalier Pitié Salpêtrière, Paris, France) in accordance with the 1964 Declaration of Helsinki and its later amendments. Written informed consent was obtained from all participants.