Five-Year Extension Results of the Phase 1 START Trial of Onasemnogene Abeparvovec in Spinal Muscular Atrophy
Onasemnogene Abeparvovec治疗脊髓性肌萎缩症的1期START试验五年扩展结果
期刊:Jama Neurology
影响因子:21.3
doi:10.1001/jamaneurol.2021.1272
Mendell, Jerry R; Al-Zaidy, Samiah A; Lehman, Kelly J; McColly, Markus; Lowes, Linda P; Alfano, Lindsay N; Reash, Natalie F; Iammarino, Megan A; Church, Kathleen R; Kleyn, Aaron; Meriggioli, Matthew N; Shell, Richard