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FDA批准Givosiran治疗急性肝卟啉症 2019.11.20
Givosiran 急性肝卟啉症 研发注册 罕见病

On November 20, 2019, the Food and Drug Administration approved givosiran (GIVLAARI, Alnylam Pharmaceuticals, Inc.) for adults with acute hepatic porphyria (AHP)【FDA批准首例治疗遗传性罕见病的疗法】 Efficacy was evaluated in ENVISION (NCT03338816)...