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Title:

Long-term efficacy and safety of nusinersen in adults with 5q spinal muscular atrophy: a prospective European multinational observational study

Document type:
Article
Author(s):
Guenther, Rene; Wurster, Claudia Diana; Brakemeier, Svenja; Osmanovic, Alma; Schreiber-Katz, Olivia; Petri, Susanne; Uzelac, Zeljko; Hiebeler, Miriam; Thiele, Simone; Walter, Maggie C.; Weiler, Markus; Kessler, Tobias; Freigang, Maren; Lapp, Hanna Sophie; Cordts, Isabell; Lingor, Paul; Deschauer, Marcus; Hahn, Andreas; Martakis, Kyriakos; Steinbach, Robert; Ilse, Benjamin; Roediger, Annekathrin; Bellut, Julia; Nentwich, Julia; Zeller, Daniel; Muhandes, Mohamad Tareq; Baum, Tobias; Koch, Jan Chri...     »
Abstract:
Background Evidence for the efficacy of nusinersen in adults with 5q -associated spinal muscular atrophy (SMA) has been demonstrated up to a period of 16 months in relatively large cohorts but whereas patients reach a plateau over time is still to be demonstrated. We investigated the efficacy and safety of nusinersen in adults with SMA over 38 months, the longest time period to date in a large cohort of patients from multiple clinical sites. Methods Our prospective, observational study included...     »
Journal title abbreviation:
Lancet Reg Health Eur
Year:
2024
Journal volume:
39
Fulltext / DOI:
doi:10.1016/j.lanepe.2024.100862
Pubmed ID:
http://view.ncbi.nlm.nih.gov/pubmed/38361750
TUM Institution:
1379; 1411; 1430; Klinik und Poliklinik für Neurologie (Prof. Hemmer)
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