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 ...