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Article History

Received: 28 October 2025

Accepted: 20 January 2026

First Online: 3 February 2026

Declarations

:

: The study conforms with the principles outlined in the Declaration of Helsinki, the Good Clinical Practice, the Geneva Declaration, the Belmont Report, and the laws and regulations applicable in Germany. The institutional review board of the Charité – Universitätsmedizin Berlin approved the study (EA2/169/22). Every human provided informed consent for participation.

: All authors and participating patients gave their consent for publication.

: HFP has received personal funding by Deutsche Gesellschaft für Muskelkranke (DGM) GmbH and Berlin Institute of Health (BIH) as well as has received personal funding, travel support, and speaker fees by Alnylam Pharmaceuticals Inc., Bayer, and AstraZeneca unrelated to this project. SM received research funding, financial reimbursement for consulting, advisory board activities, speaker fees and/or contributions to congresses by AbbVie, AstraZeneca, Novartis unrelated to this project. FM received research funding, financial reimbursement for consulting, advisory board activities, speaker fees and/or contributions to congresses by AbbVie, AstraZeneca, Novartis unrelated to this project. AM received research funding by the foundation Charité (BIH clinican scientist program). RK declares no competing interests. KH received financial reimbursement for consulting, advisory board activities, speaker fees and/or contributions to congresses and travel support to attend scientific meetings by Akcea Therapeuticals Inc., Alnylam Pharmaceuticals Inc., Amicus, AstraZeneca, Bayer, BridgeBio, GSK, Hormosan, Takeda Pharmaceutical Inc., Pfizer Pharmaceuticals Inc., Purpose Pharma and Swedish Orphan Biovitrum Inc and ViiV Healthcare GmbH. KH further received research funding by the foundation Charité (BIH clinical fellow, BIH digital accelerator), Alnylam Pharmaceuticals Inc., and Pfizer Pharmaceuticals.