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Article History

Received: 26 July 2021

Accepted: 10 October 2021

First Online: 20 October 2021

Declarations

:

: The clinical protocol was approved by the ethics committees of the Universities of Heidelberg (S-554/2018), Hamburg (MC-265/19) and Munich (18-269) for sample collection of SMA and DMD patients, and the ethics committee of the State Medical Council of Baden Württemberg for sample collection of healthy controls (F-2013-006#A). All patients and their legal guardians enrolled in this study provided signed informed consent.

: Not applicable.

: Afshin Saffari received partial funding for the study through a Sponsor Research Agreement with Biogen Inc. He received travel grants from Biogen Inc., which manufactures therapies for SMA. Claire Cannet has nothing to report. Astrid Blaschek has nothing to report. Andreas Hahn has nothing to report. Georg F. Hoffmann received a grant for the establishment of prediction models for screened newborns with SMA from the Dietmar Hopp Foundation, St. Leon-Rot, Germany. Jessika Johannsen received advisory board and/or speaker honoraria and financial support for conference attendances from Avexis, Biogen, Roche, PTC and Sarepta. Romy Kirsten has nothing to report. Musa Kockaya has nothing to report. Stefan Kölker received a grant for the establishment of prediction models for screened newborns with SMA from the Dietmar Hopp Foundation, St. Leon-Rot, Germany. Wolfgang Müller-Felber reports personal fees from Avexis (Advisory baord, Talks), personal fees from Biogen (Advisory baord, Talks), personal fees from PTC (Advisory baord, Talks), personal fees from Sarepta (Advisory baord, Talks), personal fees from Roche (Advisory board) and personal fees from Sanofi-Aventis (Advisory baord, Talks). Andreas Roos has nothing to report. Hartmut Schäfer has nothing to report. Ulrike Schara received advisory board and/or speaker honoraria from Avexis, Biogen, Roche, PTC, Santhera, Sarepta, Pfizer, Alexion and Sanofi. Manfred Spraul has nothing to report. Friedrich K. Trefz has nothing to report. Katharina Vill has nothing to report. Wolfgang Wick has nothing to report. Markus Weiler received advisory board and/or speaker honoraria and financial support for conference attendances from Akcea Therapeutics, Alnylam Pharmaceuticals, Biogen, and Pfizer, and advisory board and consultant honoraria from Hoffmann-La Roche. Jürgen G. Okun has nothing to report. Andreas Ziegler received partial funding for the study through a Sponsor Research Agreement with Biogen Inc. He received advisory board and/or speaker honoraria and financial support for conference attendances from Avexis, Biogen, Roche, PTC, and Santhera.