Efficacy and Safety of Gene Therapy with Onasemnogene Abeparvovec in Children with Spinal Muscular Atrophy in the D-A-CH-region: a Population-Based Observational Study
LANCET REGIONAL HEALTH-EUROPE（2024）
关键词
Spinal muscular atrophy,Gene addition therapy,SMA,Onasemnogene abeparvovec,Gene therapy,Zolgensma
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