Ethics approval and consent to participate The local ethics committees of all participating sites approved the study and all patients and controls signed written informed consent.
Consent for publication Not applicable.
Availability of data and materials The datasets used and/or analyzed during the current study are available from the corresponding author on reasonable request.
Competing interests MF reports non-financial support from Biogen outside the submitted work.
PS reports no disclosures.
CDW has received honoraria from Biogen as an advisory board member and for lectures and as a consultant and advisory board member from Hoffmann‑La Roche. She also received travel expenses from Biogen.
OSK has received honoraria as a speaker/consultant and/or funding for travel expenses from the German Neuromuscular Society “Deutsche Gesellschaft fuer Muskelkranke” (DGM e.V.), Novartis, Biogen GmbH, Biermann Verlag GmbH, MK+S - Medizin, Kommunikation & Service GmbH, and the Jain Foundation, outside the submitted work; and research support from the DGM e.V., outside the submitted work.
AO has received honoraria from Biogen for lectures.
SP has received grants from the German Neuromuscular Society, the Federal Ministry of Education and Research, the German Israeli Foundation for Scientific Research and Development, and the EU Joint Programme for Neurodegenerative Disease Research; and other support from Cytokinetics, Desitin Pharma, Biogen, Novartis, and Teva outside of the submitted work.
JCK has received payment for consultation and advisory board participation from Biogen, Hoffmann‑La Roche and AveXis.
KR has no conflicts of interest to declare that are relevant to the content of this article.
BF has no conflicts of interest to declare that are relevant to the content of this article.
ACL has received personal fees from AB Science, Biogen, Cytokinetics, GlaxoSmithKline, Orion Pharma, Novartis, Tau Rx Therapeutics, Teva, Mitsubishi, and Hoffmann‑La Roche outside of the submitted work.
MO has no conflicts of interest to declare that are relevant to the content of this article.
AH received royalties from BIOGEN and DESITIN as an advisory board member.
RG has received honoraria from Biogen as an advisory board member and for lectures and as
a consultant and advisory board member from Hofmann-La Roche. He also received travel expenses and research support from Biogen
Funding No funding was received for conducting this study.
Authors’ contributions All authors contributed to the acquisition of data and revised the manuscript for intellectual content. MF, PS and RG analyzed and interpreted the data and prepared the original draft. AH and RG concepted and designed the study. RG did supervision and project administration.
Acknowledgements We thank all patients for participation. CDW is supported by the Charcot-Stiftung. OSK receives academic research support from the Hannover Medical School Young Faculty Program, 2018-20. OSK received academic research support from the Hannover Medical School Young Faculty Program, 2018–2020; currently, OSK is funded by the “Ellen-Schmidt-Program—Habilitationsfoerderung fuer Wissenschaftlerinnen”, Hannover Medical School (2021). AH is supported by the Hermann und Lilly Schilling-Stiftung für medizinische Forschung im Stifterverband. Individual centers received funding from SMArtCARE and Biogen, who both had no role in study design, data collection, data analysis, data interpretation, or writing of the report.