Nusinersen in adults with 5q spinal muscular atrophy: a non-interventional, multicentre, observational cohort study
2020 | journal article; research paper. A publication with affiliation to the University of Göttingen.
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Nusinersen in adults with 5q spinal muscular atrophy: a non-interventional, multicentre, observational cohort study
Hagenacker, T.; Wurster, C. D.; Günther, R.; Schreiber-Katz, O.; Osmanovic, A.; Petri, S. & Weiler, M. et al. (2020)
The Lancet. Neurology, 19(4) pp. 317-325. DOI: https://doi.org/10.1016/S1474-4422(20)30037-5
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Details
- Authors
- Hagenacker, Tim; Wurster, Claudia D.; Günther, René; Schreiber-Katz, Olivia; Osmanovic, Alma; Petri, Susanne; Weiler, Markus; Ziegler, Andreas; Kuttler, Josua; Koch, Jan C; Schneider, Ilka; Wunderlich, Gilbert; Schloss, Natalie; Lehmann, Helmar C.; Cordts, Isabell; Deschauer, Marcus; Lingor, Paul; Kamm, Christoph; Stolte, Benjamin; Pietruck, Lena; Totzeck, Andreas; Kizina, Kathrin; Mönninghoff, Christoph; von Velsen, Otgonzul; Ose, Claudia; Reichmann, Heinz; Forsting, Michael; Pechmann, Astrid; Kirschner, Janbernd; Ludolph, Albert C.; Hermann, Andreas; Kleinschnitz, Christoph
- Abstract
- Nusinersen is approved for the treatment of 5q spinal muscular atrophy of all types and stages in patients of all ages. Although clinical trials have shown improvements in motor function in infants and children treated with the drug, data for adults are scarce. We aimed to assess the safety and efficacy of nusinersen in adults with 5q spinal muscular atrophy.
- Issue Date
- 2020
- Journal
- The Lancet. Neurology
- ISSN
- 1474-4422
- eISSN
- 1474-4465
- Language
- English
