Onasemnogene Abeparvovec Gene Therapy for Symptomatic Infantile-Onset Spinal Muscular Atrophy in Patients with Two Copies of SMN2 (STR1VE): an Open-Label, Single-Arm, Multicentre, Phase 3 TrialJohn W. Day,Richard S. Finkel,Claudia A. Chiriboga,Anne M. Connolly,Thomas O. Crawford,Basil T. Darras,Susan T. Iannaccone,Nancy L. Kuntz,Loren D. M. Pena,Perry B. Shieh,Edward C. Smith,Jennifer M. Kwon,Craig M. Zaidman,Meredith Schultz,Douglas E. Feltner,Sitra Tauscher-Wisniewski,Haojun Ouyang,Deepa H. Chand,Douglas M. Sproule,Thomas A. Macek,Jerry R. MendellLancet neurology（2021）引用 195|浏览56暂无评分AI 理解论文溯源树样例生成溯源树，研究论文发展脉络Chat Paper正在生成论文摘要
