Efficacy and safety of risdiplam in adults with 5q-associated spinal muscular atrophy: a nationwide observational cohort study in Austria.
Keritam O, Erdler M, Fasching B, Zulehner G, Rath J, Krenn M, Waldhör T, Gruber VA, Langweil N, Kiss C, Griedl TA, Gold V, Wanschitz J, Hotter A, Kleinveld VEA, Horlings CGC, Erber A, Schernhammer E, Troger J, Grinzinger S, Müller P, Langenscheidt D, Rappold M, Wiesenhofer A, Gosk-Tomek M, Knipp F, Mahal S, Bernert G, Baumann M, Zimprich F, Topakian R, Eggers C, Quasthoff S, Löscher W, Cetin H.
Keritam O, et al.
EClinicalMedicine. 2025 Sep 26;88:103536. doi: 10.1016/j.eclinm.2025.103536. eCollection 2025 Oct.
EClinicalMedicine. 2025.
PMID: 41181835
Free PMC article.