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Article History

Received: 26 August 2025

Accepted: 13 November 2025

First Online: 9 December 2025

Declarations

:

: Not applicable.

: Not applicable.

: RQ: Honoraria for lectures: PTCbio, Sarepta, Sanofi genzyme, Biogen, Santhera. Participation in data monitoring committees and advisory boards: Roche, TRiNDS, PTCbio, Sarepta, Santhera. PI in clinical trials for PTCbio, Edgewise, Dynacure, unrelated to the current work. TS is recipient of research grant from Pfizer, and honoraria received for lectures or consultancy from Biogen, Novartis, PTC Therapeutics, Sarepta Therapeutics, Pfizer, Roche, and Italfarmaco, unrelated to the current work. KGC received speaker/advisory board honoraria from Alexion, Alnylam, Amgen, Amicus, argenx, AstraZeneca, Biogen, Edgewise, Ipsen Pharma, Janssen Pharmaceutics, Lupin, Pfizer, Roche, Sanofi-Genzyme, UCB; and is recipient of research funding by CSL Behring, Alnylam, Pfizer, Sanofi-Genzyme, Vertex, Biogen and Roche, unrelated to the current work. The other authors declare no conflict of interest. The funders had no role in the design of the study; in the collection, analyses, or interpretation of data; in the writing of the manuscript; or in the decision to publish the results.