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庞佩病:当前状态和未来的治疗方法

Margot Richards1, William H Frishman1,2

  • 1New York Medical College, Valhalla, NY.

Cardiology in review
|July 2, 2025
PubMed
概括

庞培病是一种遗传性疾病,目前使用酶替代疗法 (ERT) 治疗. 基因疗法为庞培病提供了一个有前途的新途径,显示出安全性和持续治疗的潜力.

关键词:
庞贝病是庞贝病的一种疾病.酶替代疗法是一种酶替代疗法.基因治疗的基因疗法糖原储存疾病 糖原储存疾病

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