High-dose nusinersen for spinal muscular atrophy: a phase 3 randomized trial
高剂量诺西那生治疗脊髓性肌萎缩症:一项3期随机试验
期刊:Nature Medicine
影响因子:50
doi:10.1038/s41591-025-04193-6
Finkel, Richard S; Crawford, Thomas O; Mercuri, Eugenio; Sumner, Charlotte J; Garcia Romero, Maria Del Mar; Day, John W; Montes, Jacqueline; Sun, Peng; Tichler, Ben; Paradis, Angela D; Boesch, Emily; Inra, Jennifer; Littauer, Ross; Sohn, Jihee; Monine, Michael; Gambino, Giulia; Foster, Richard; Farewell, Raechel; Fradette, Stephanie