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Authors
Mendelsohn, Daniel H.
Rosenbohm, Angela
Güttsches, Anne-Katrin
Kornblum, Cornelia
Knop, Karl Christian
Fangerau, Tanja
Nguyen-Younossi, Nam
Shahyrova, Guljan
Garcia-Angarita, Natalia
Schoser, Benedikt
Wenninger, Stephan https://orcid.org/0000-0001-8407-3642
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Funding
Funding for this research was provided by:
Klinikum der Universität München
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Article History
Received: 19 November 2025
Revised: 3 February 2026
Accepted: 5 February 2026
First Online: 14 February 2026
Declarations
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: All authors report no disclosures regarding this study. Outside of this context, SW has received research grant from the DGM—Deutsche Gesellschaft fuer Muskelkranke e.V. He has served on advisory boards for Alexion Pharma, CSL Behring, and Sanofi Genzyme GmbH. He received funding for travel or speaker honoraria from Sanofi-Aventis Germany GmbH; SH Glykogenose Gesellschaft; AbbVie Germany GmbH; Recordati Pharma GmbH; CSL Behring GmbH; Alexion Pharma GmbH; Desitin Germany; Akcea GmbH. Outside of this context, BS has received unrestricted research grants from AMDA Foundation, Amicus Therapeutics, Inc., EU Horizon programs COMPASS, PaLaDIn, Entry-DM, and Roche Diagnostics; speakers’ honoraria from Alexion Pharmaceuticals Inc., Amicus Therapeutics, Inc., Argenx, Dyne, Kedrion; and has also been a scientific adviser for Amicus Therapeutics, Inc., Alexion Pharmaceuticals Inc., Astellas, Avidity, Dyne, Johnson&Johnson, and Sanofi. He declares no stocks or shares. DHM has received funding for travel and a speaker honorarium from. AR has received research grant from the DGM—Deutsche Gesellschaft fur Muskelkranke e.V. She has received funding for travel or speaker honoraria from Sanofi-Aventis Germany GmbH; Alexion Pharma GmbH; Amicus Therapeutics GmbH, Argenx Germany GmbH, Hormosan Pharma GmbH, Alnylam Germany GmbH, Fulcrum Therapeutics Cambridge, MA. Outside of this context, AG received remuneration for lecturing activity from Sanofi-Aventis Germany GmbH. She received remuneration for participation as a consultant in the advisory board from Amicus Therapeutics GmbH. Outside of this context, CK has received travel funding and/or speaker honoraria from Amicus Therapeutics, Argenx, Chiesi, Fulcrum Therapeutics, Novartis, Sanofi Genzyme, Santhera, Sarepta Therapeutics, UCB, and served as advisory board member and/or principal investigator for Amicus Therapeutics, Chiesi, Fulcrum Therapeutics, Hormosan, Omeicos, Reneo Pharmaceuticals, Roche Pharma AG, Sanofi Genzyme, Stealth BioTherapeutics, Symptoma, Tisento. The other authors have no conflicts of interest to declare.
