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Article History

Received: 5 March 2026

Accepted: 16 April 2026

First Online: 24 April 2026

Declarations

:

: K.H. received financial reimbursement for consulting, advisory boards, educational activities and travel support by Akcea, Alnylam, Amicus, AstraZeneca, Bayer, Bridge Bio, GSK, Hormosan, SOBI, Pfizer, ViiV and Takeda. She also received research funding by Alnylam, Pfizer and the foundation Charité (BIH clinical fellow, BIH digital accelerator). Nothing was related to this project. S.S. reports scientific grants from Innovation Committee at the Federal Joint Committee (G-BA), Pfizer Pharma GmbH, and Apple Inc.; Honoraria for lectures from Novartis Pharma GmbH, AstraZeneca GmbH, Pfizer Pharma GmbH, Alnylam Netherlands B.V., and Bayer Vital GmbH; travel fees from Pfizer Pharma GmbH, Alnylam Netherlands B.V., AstraZeneca GmbH, and Boehringer Ingelheim; Advisory fees from Novartis Pharma GmbH, Pfizer Pharma GmbH, Bayer Vital GmbH, Alnylam Netherlands B.V., and Bayer Vital GmbH. V.R.L. has received speaker fees from Johnson&Johnson and Sanofi unrelated to this project. G.B. received personal funding by Alnylam Pharmaceuticals Inc. H.P. has received personal funding, travel support, and speaker fees by Alnylam Pharmaceuticals Inc. HP also received personal funding by Deutsche Gesellschaft für Muskelkranke (DGM) GmbH and Berlin Institute of Health (BIH) unrelated to this project. D.M. received research funding from Pfizer Pharma GmbH, lecture fees from Pfizer Pharma GmbH and Alnylam Pharmaceuticals Inc., and financial reimbursement for consulting and advisory board activities from Bayer Vital GmbH. I.M. has received research grants from Pfizer Pharmaceuticals and Sanofi; and has received lecture fee and financial reimbursement for advisory board activities from Sanofi, Takeda, Pfizer Pharmaceuticals, BridgeBio, Bayer, Doctorflix, Amicus, and Chiesi Farmaceutici S.p.A outside the submitted work. Remaining authors declare no conflicts of interest.

: The study obtained ethics approval from the Ethics Committee of Charité – Universitätsmedizin Berlin, Campus Charité Mitte, under application numbers EA1/014/20 and EA4/235/20. All participants gave informed consent before taking part. All patient data were fully pseudonymized, and individuals are not identifiable. The research publication is granted via the signed patient consent form and all participants gave informed consent before taking part.

: All authors declare their consent for publication.