Intrathecal onasemnogene abeparvovec for treatment-experienced patients with spinal muscular atrophy: a phase 3b, open-label trial
鞘内注射onasemnogene abeparvovec治疗既往接受过治疗的脊髓性肌萎缩症患者:一项3b期开放标签试验
期刊:Nature Medicine
影响因子:50
doi:10.1038/s41591-025-04119-2
Kwon, Jennifer M; Munell, Francina; Le Goff, Laure; Yuge, Kotaro; Kato, Tamaki; Cances, Claude; De Waele, Liesbeth; Woodcock, Ian R; Mercuri, Eugenio M; Proud, Crystal M; Darras, Basil T; Hayes, Leslie H; Oskoui, Maryam; Visootsak, Jeannie; Williams, Gemma; Ilić, Andreja; Yang, Lina; van der Pol, W Ludo