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FDA批准首例治疗遗传性罕见病的疗法 2019.11.20

For Immediate Release November 20, 2019 Today, the U.S. Food and Drug Administration granted approval to Givlaari (givosiran) for the treatment of adult patients with acute hepatic porphyria【FDA批准Givosiran治疗急性肝卟啉症】, a genetic disorder res...

FDA批准Givosiran治疗急性肝卟啉症 2019.11.20

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)...