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Authors
Leibrock, Berenike
Landfeldt, Erik
Hussong, Justine
Huelle, Tabea
Mattheus, Hannah
Thiele, Simone
Walter, Maggie C.
Zemlin, Michael
Moehler, Eva
Dillman, Ullrich
Abner, Sophia
Flotats-Bastardas, Marina https://orcid.org/0000-0001-7696-4808
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Funding
Funding for this research was provided by:
Initiative SMA-Gemeinsam für eine Therapie
Deutsche Forschungsgemeinschaft
Universitätsklinikum des Saarlandes und Medizinische Fakultät der Universität des Saarlandes
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Article History
Received: 15 October 2022
Accepted: 6 February 2023
First Online: 21 February 2023
Declarations
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: The study was approved by the regional Ethics Committee from the Saarland Medical Association on 04.02.2020 with protocol number 09/20. All adult participants and respectively the caregiver for minors provided informed consent to participate in the study.
: Not applicable.
: Dr. Marina Flotats-Bastardas has received consultant fees from Roche and Biogen. Dr. Landfeldt and Ms. Abner are employees of IQVIA, a contract research organization. Dr. Maggie C. Walter has served on advisory boards for Avexis, Biogen, Novartis, Pfizer, Roche, Santhera, Sarepta, Pharnext, PTC Therapeutics, Ultragenyx, Wave Sciences, received funding for Travel or Speaker Honoraria from Avexis, Biogen, PTC Therapeutics, Ultragenyx, Santhera, Sarepta, and worked as an ad-hoc consultant for AskBio, Audentes Therapeutics, Avexis, Biogen Pharma GmbH, Fulcrum Therapeutics, GLG Consult, Guidepoint Global, Gruenenthal Pharma, Novartis, Pharnext, PTC Therapeutics, Roche. Simone Thiele has received financial support for advisory services from PTC. The other authors declare that there is no conflict of interest.
