Intrathecal onasemnogene abeparvovec in treatment-naive patients with spinal muscular atrophy: a phase 3, randomized controlled trial
鞘内注射onasemnogene abeparvovec治疗初治脊髓性肌萎缩症患者：一项3期随机对照试验
期刊:Nature Medicine
影响因子:50
doi:10.1038/s41591-025-04103-w
Proud, Crystal M; Vũ, Dũng Chí; Wilmshurst, Jo M; Sanmaneechai, Oranee; Gulati, Sheffali; Xiong, Hui; Moreno, Hugo C; Tay, Stacey Kiat Hong; Thong, Meow-Keong; Born, Alfred Peter; Banzzatto Ortega, Adriana; Jong, Yuh-Jyh; Al-Muhaizea, Mohammad A; Lee, Anna W; Visootsak, Jeannie; Tauscher-Wisniewski, Sitra; Alecu, Iulian; Parlikar, Rutvick; Finkel, Richard S
