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Updated: May 6, 2026

In Vitro Enzyme Measurement to Test Pharmacological Chaperone Responsiveness in Fabry and Pompe Disease
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Published on: December 20, 2017

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使用Fabry Outcome和Gaucher Outcome调查的数据,对自我使用的酶替代疗法的安全性分析

Shoshana Revel-Vilk1,2, Uma Ramaswami3, Guillem Pintos-Morell4

  • 1Gaucher Unit and Pediatric Hematology/Oncology Unit, The Eisenberg R&D Authority, Shaare Zedek Medical Center, Jerusalem, Israel.

Orphanet journal of rare diseases
|March 29, 2025
PubMed
概括

对法布里和高沙病的酶替代疗法 (ERT) 的自我管理是安全的. 与医疗保健提供者支持的输液相比,自我给药的阿尔法甲基酶或阿尔法黄糖酶患者的输液反应较少.

关键词:
阿尔法阿格尔西达酶的使用酶替代疗法是一种酶替代疗法.这就是FOSOS.布里 布里 布里 布里 布里这就是GOS.的左撇子 (Gaucher Gaucher) 是一个非常有价值的人.在家进行家庭治疗.安全的安全的安全的安全的安全.自主管理的自治.维拉葡萄糖酶αα的使用

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