暴涨247%,uniQure基因疗法亨廷顿病关键临床成功;盟科药业第一大股东反对多项提案

Ofweek维科网
Sep 26

创新药暴力美学。9月24日,uniQure宣布,其基因疗法AMT-130用于治疗亨廷顿病的关键性 I/II 期研究获得积极顶线数据。该研究达到了预设的主要终点,与倾向评分匹配的外部对照组相比,高剂量 AMT-130 在 36 个月时通过复合亨廷顿病评定量表(cUHDRS)测量显示出统计学上显著的疾病进展减缓。该研究还达到了一个关键的次要终点,即与倾向评分匹配的外部对照组相比,在 36 个月时通过总...

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