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Article History

Received: 2 May 2023

Revised: 14 June 2023

Accepted: 6 July 2023

First Online: 17 July 2023

Declarations

:

: All authors report no disclosures regarding this study. Outside of this context, SW and KG have received a research grant from the DGM—Deutsche Gesellschaft für Muskelkranke e.V. KG received travel or speaker Honoraria from Sanofi-Aventis Germany GmbH, Amicus Therapeutics and Kedrion. SW has served on advisory boards for Alexion Pharma, CSL Behring and Sanofi Genzyme GmbH. In addition, he received funding for travel or speaker Honoraria from Sanofi-Aventis Germany GmbH; Amicus Therapeutics; 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 served on advisory boards for Amicus, Argenex, Astellas, and Sanofi; he has undertaken contracted available research for Amicus, Astellas, Roche, DFG, Horizon 2022, AMDA USA, Marigold foundation CA; and has received speaker honoraria from Alexion and Kedrion.

: All procedures performed in this study were in accordance with the ethical standards of the institutional and/or national research committee and with the 1964 Helsinki declaration and its later amendments or comparable ethical standards. The study was approved by the ethics committee of the LMU Klinikum, Project No. 19-613. In addition, the protocol was registered on a public clinical trials registry (ClinicalTrials.gov Identifier NCT04411732). All persons gave their informed consent prior to their inclusion in the study.