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 ...
JEWELFISH: 24-month results from an open-label study in non-treatment-naïve patients with SMA receiving treatment with risdiplam Abstract 摘要 Risdiplam is a once-daily oral, survival of motor neuron 2 (SMN2) splicing modifier approved for the treatment of spinal muscular atro ...