ClinicalTrials.gov · 小核酸药物临床试验· Biogen·· 2020-07-27AI 评分15
Nusinersen（BIIB058）用于既往接受Onasemnogene Abeparvovec治疗的SMA患儿研究（RESPOND）
A Study to Learn About the Effect of Nusinersen (BIIB058) Given as Injections to Children With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec (RESPOND)
AI 导读
一项研究将评估Nusinersen（BIIB058）对3岁以下、既往接受过基因治疗Onasemnogene Abeparvovec但仍有疾病相关健康问题的SMA患儿的运动功能影响，主要终点为治疗后HINE第2部分运动里程碑评分。
来源：ClinicalTrials.gov · 小核酸药物临床试验 · clinicaltrials.gov