$uniQure NV (QURE.US)$ uniQure Announces Orphan Drug Designation Granted to AMT-191 for the Treatment of Fabry Disease uniQure N.V. (NASDAQ: QURE) announced that the U.S. FDA has granted Orphan Drug Designation to AMT-191, their investigational gene therapy for Fabry disease. This follows the dosing of the first patient in a U.S. multi-center, open-label Phase I/IIa trial in August 2024. AMT-191 is a one-time intravenously administered AAV5-based gene therapy targeting the liver to produce G...
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我选择了错误的Curi!哈哈,但两者都不错。
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uniQure Announces Orphan Drug Designation Granted to AMT-191 for the Treatment of Fabry Disease
uniQure N.V. (NASDAQ: QURE) announced that the U.S. FDA has granted Orphan Drug Designation to AMT-191, their investigational gene therapy for Fabry disease. This follows the dosing of the first patient in a U.S. multi-center, open-label Phase I/IIa trial in August 2024. AMT-191 is a one-time intravenously administered AAV5-based gene therapy targeting the liver to produce G...
uniQure宣布AMt-191用于法布病治疗的I/IIa临床试验已开始给第一个患者进行剂量投放。
uniQure N.V.(NASDAQ:QURE)已开始对AMt-191进行法布瑞病治疗的I/IIa期临床试验的剂量给药。在美国进行的多中心、开放标签试验将评估两个剂量递增队列的安全性、耐受性和早期疗效迹象。AMt-191是基于AAV5的基因疗法,可以传递α半乳糖苷酶(GLA)转基因至人体细胞内。
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