Respiratory outcomes of onasemnogene abeparvovec treatment for spinal muscular atrophy: national real-world cohort study
脊髓性肌萎缩症患者接受onasemnogene abeparvovec治疗的呼吸系统结局:一项全国真实世界队列研究
期刊:European Journal of Pediatrics
影响因子:2.6
doi:10.1007/s00431-024-05886-9
Lavie, Moran; Rochman, Mika; Armoni Domany, Keren; Golan Tripto, Inbal; Be'er, Moria; Besor, Omri; Sagi, Liora; Aharoni, Sharon; Ginsberg, Mira; Noyman, Iris; Levine, Hagit