(以下内容从华福证券《生物医药行业定期:看好创新药产业趋势,关注ESMO和医保谈判》研报附件原文摘录)臂的亨廷顿基因上的CAG三核苷酸异常扩增突变所致,通常在发病后15-20年内死亡。2025年9月24日,基因治疗公司uniQureN.V.宣布了基因治疗药物AMT-130治疗亨廷顿舞蹈症的关键I/II期研究的积极顶线数据,主要终点复合统一亨廷顿病评定量表(cUHDRS)显示高剂量AMT-130显示...
Source Link(以下内容从华福证券《生物医药行业定期:看好创新药产业趋势,关注ESMO和医保谈判》研报附件原文摘录)臂的亨廷顿基因上的CAG三核苷酸异常扩增突变所致,通常在发病后15-20年内死亡。2025年9月24日,基因治疗公司uniQureN.V.宣布了基因治疗药物AMT-130治疗亨廷顿舞蹈症的关键I/II期研究的积极顶线数据,主要终点复合统一亨廷顿病评定量表(cUHDRS)显示高剂量AMT-130显示...
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