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Article History

Received: 9 December 2020

Accepted: 16 March 2021

First Online: 31 March 2021

Declarations

:

: The local ethics committee of the participating universities (Ludwig-Maximilians-University of Munich, University of Munster and University of Essen, project no. 18–269) has approved the study.

: Informed consent for prospective follow-up was obtained from the participating families except from those who did not make appointments from the start or did not take up the treatment options. For patients without written consent, which is congruent with those who did not follow the recommended procedure, no further standardized examinations were carried out. For these patients, we report on the natural course of events known to us.

: All authors have indicated that they don’t have any non-financial conflicts of interest. Financial Disclosure: Astrid Blaschek, Bernhard Olgemöller, Erik Harms, Katja Eggermann and Uta Nennstiel have nothing to disclose. Marc Becker is the owner of a commercial entity (Laboratory Becker and colleagues MVZ GbR, Führichstraße 70, 81871 Munich, Germany). Siegfried Burggraf, Wulf Röschinger, Jürgen Durner, Ludwig Czibere are employed at a commercial entity (Laboratory Becker and colleagues MVZ GbR, Führichstraße 70, 81871 Munich, Germany). Dieter Gläser is the co-owner of a commercial entity (Genetikum®, Wegenerstr. 15, 89231 Neu-Ulm, Germany). Katharina Vill has received travel expenses and speaker fees from Biogen. Oliver Schwartz has served as a member of a scientific advisory board for Avexis and received travel expenses and speaker fees from Biogen. Heike Kölbel has served as a member of a scientific advisory board for Avexis and received travel expenses and speaker fees from Biogen and Sanofi-Aventis. Ulrika Schara has served as a member of a scientific advisory board and data safety monitoring board for Biogen, Avexis and Novartis and received speaker fees from Biogen, Avexis, PTC and Sanofi-Aventis. Brunhilde Wirth has served as a member of a scientific advisory board and data safety monitoring board for SMA Europe and received travel expenses and speaker fees from Biogen. Wolfgang Müller-Felber has served as a member of a scientific advisory board for Biogen, Avexis, PTC, Sanofi-Aventis, Roche and Cytokinetics and received travel expenses and speaker fees from Biogen, Avexis, PTC and Sanofi-Aventis.