Efficacy and safety of risdiplam in adults with 5q-associated spinal muscular atrophy: a nationwide observational cohort study in Austria
利司帕林治疗5q相关脊髓性肌萎缩症成人患者的疗效和安全性：一项奥地利全国性观察性队列研究
期刊:EClinicalMedicine
影响因子:10
doi:10.1016/j.eclinm.2025.103536
Keritam, Omar; Erdler, Marcus; Fasching, Bernhard; Zulehner, Gudrun; Rath, Jakob; Krenn, Martin; Waldhör, Thomas; Gruber, Victoria Anna; Langweil, Nadine; Kiss, Christian; Griedl, Theresa Antonia; Gold, Valeriu; Wanschitz, Julia; Hotter, Anna; Kleinveld, Vera E A; Horlings, Corinne G C; Erber, Astrid; Schernhammer, Eva; Troger, Johannes; Grinzinger, Susanne; Müller, Petra; Langenscheidt, Dieter; Rappold, Mika; Wiesenhofer, Anna; Gosk-Tomek, Magdalena; Knipp, Florian; Mahal, Simone; Bernert, Günther; Baumann, Matthias; Zimprich, Fritz; Topakian, Raffi; Eggers, Christian; Quasthoff, Stefan; Löscher, Wolfgang; Cetin, Hakan
