Real-World Outcomes of Risdiplam in 5q Spinal Muscular Atrophy Over 30 Months in Adolescents and Adults: A Multicentre, Prospective, Observational SMArtCARE Study

SSRN Electronic Journal 2026 Preprint Open access

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Authors: Maggie Christine Walter, Markus Weiler, Otgonzul von Velsen, Anika Hüsing, Matthias Baumann, Caroline Gertrud Bergner, Astrid Blaschek, Hakan Çetin, Marcus Deschauer, Astrid Eisenkölbl, René Günther, Andreas Hahn
+39 more Miriam Hiebeler, Elke Hobbiebrunken, Dorothea Holzwarth, Ralf A. Husain, Jessika Johannsen, Birgit Kauffmann, Omar Keritam, Janbernd Kirschner, Felix Kleefeld, Christoph Kleinschnitz, Johannes Koch, Heike Kölbel, Wolfgang N. Löscher, Alexander Mensch, Andreas Merkenschlager, Gerd Meyer zu Hörste, Christoph Neuwirth, Astrid Pechmann, Susanne Petri, Veronika Pilshofer, Nina Rademacher, Christof Reihle, Annekathrin Rödiger, Georges Sam, Bertold Schrank, Gudrun Schreiber, Oliver Schwartz, Anette Schwerin-Nagel, Christian Stadler, Meike Steinbach, Regina Trollmann, Matthias Türk, Željko Uzelac, Katharina Vill, Gilbert Wunderlich, Daniel Zeller, SMArtCARE study group, Hanns Lochmüller, Tim Hagenacker
DOI: 10.2139/ssrn.6167932
Cited by: 0

Fields of Study & Subjects

Neurogenetic and Muscular Disorders Research Lysosomal Storage Disorders Research Botulinum Toxin and Related Neurological Disorders

Publication details

Journal / source
SSRN Electronic Journal
Publication date
1 January 2026
Document type
Preprint