Efficacy and safety of gene therapy with onasemnogene abeparvovec in children with spinal muscular atrophy in the D-A-CH-region: a population-based observational study
一项基于人群的观察性研究：在DA-CH区域患有脊髓性肌萎缩症的儿童中，使用onasemnogene abeparvovec进行基因治疗的疗效和安全性
期刊:Lancet Regional Health-Europe
影响因子:13
doi:10.1016/j.lanepe.2024.101092
Weiß, Claudia; Becker, Lena-Luise; Friese, Johannes; Blaschek, Astrid; Hahn, Andreas; Illsinger, Sabine; Schwartz, Oliver; Bernert, Günther; Hagen, Maja von der; Husain, Ralf A; Goldhahn, Klaus; Kirschner, Janbernd; Pechmann, Astrid; Flotats-Bastardas, Marina; Schreiber, Gudrun; Schara, Ulrike; Plecko, Barbara; Trollmann, Regina; Horber, Veronka; Wilichowski, Ekkehard; Baumann, Matthias; Klein, Andrea; Eisenkölbl, Astrid; Köhler, Cornelia; Stettner, Georg M; Cirak, Sebahattin; Hasselmann, Oswald; Kaindl, Angela M; Garbade, Sven F; Johannsen, Jessika; Ziegler, Andreas
