Onasemnogene abeparvovec 基因疗法治疗症状性婴儿型脊髓性肌萎缩症 1 型 Summary 摘要 Background Spinal muscular atrophy is a rare, autosomal recessive, neuromuscular disease caused by biallelic loss of the survival motor neuron 1 (SMN1) gene, resulting in motor neuron dysfunc ...
Onasemnogene abeparvovec 基因疗法治疗症状性婴儿型脊髓性肌萎缩症 1 型 Summary 摘要 Background Spinal muscular atrophy type 1 is a motor neuron disorder resulting in death or the need for permanent ventilation by age 2 years. We aimed to evaluate the safety and efficacy of ...
治疗 SMA 罕见病的孤儿药 Onasemnogene Abeparvovec (Zolgensma ® ) 的真实世界安全性数据：基于 EMA 不良事件报告系统的药物警戒研究 Abstract 摘要 The recent introduction of the innovative therapy, onasemnogene abeparvovec (Zolgensma®), has revolutionized the spinal muscular atr ...
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