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治疗晚发氏病的新进展:叙述性综述

Misha Khan1, Zainab Awan2, Eesha Asghar Ali2

  • 1Department of Medicine and Surgery, Liaquat National Hospital and Medical College, National Stadium Road, Karachi, 74800, Pakistan. mishashabbir39@gmail.com.

European journal of pediatrics
|December 24, 2025
PubMed
概括

对于晚发佩病 (LOPD) 的酶替代疗法 (ERT) 选择包括阿尔古古西酶α,阿瓦古西酶α和西帕古西酶α + miglustat. 阿瓦葡萄糖酶阿尔法在LOPD管理中显示出卓越的疗效,副作用较少.

关键词:
自体逆行性疾病 自体逆行性疾病酶替代疗法替代方案 酶替代疗法替代方案晚期发病的庞贝病.治疗转换治疗转换三个S标准的标准.第二种类型的糖原储存库.

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