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Updated: Jun 26, 2025

In Vitro Enzyme Measurement to Test Pharmacological Chaperone Responsiveness in Fabry and Pompe Disease
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对酶替代疗法的首次人体临床试验的临床药理考虑

Sydney Stern1, Jie Wang1, Ruo-Jing Li1

  • 1Office of Clinical Pharmacology, Office of Translational Science, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland, USA.

Journal of inherited metabolic disease
|May 13, 2024
PubMed
概括

本综述分析了支持20个生物许可申请 (BLA) 的酶替代疗法 (ERT) 药物的第一人 (FIH) 协议. 大多数试验优先考虑安全性,耐受性和生物标志物评估,为未来的ERT开发提供信息.

关键词:
临床试验临床试验临床试验临床试验临床试验药物开发是药物的发展.酶替代疗法是一种酶替代疗法.人类中的第一位.罕见病是一种罕见的疾病.

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