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A 53-week, open-label phase IIIb study of velaglucerase alfa in Chinese patients with type 1 Gaucher disease: Safety, efficacy, and pharmacokinetics

Background: Velaglucerase alfa is approved in China for treating type 1 Gaucher disease (GD1), but data on its use in Chinese pediatric and adult patients are limited. This study evaluated the safety, efficacy, and pharmacokinetics of velaglucerase alfa in Chinese patients with GD1. Methods: This wa...

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Hoofdauteurs: Lihui Zhang, Xue-Qun Luo, Yongjun Fang, Fengkui Zhang, Yan Meng, Xiaoping Luo, Liansheng Zhang, Zhengqing Qiu, Xiao Ge, Jaco Botha, Jenny Zhan, Tianyou Wang
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: Elsevier 2026-06-01
Reeks:Molecular Genetics and Metabolism Reports
Onderwerpen:
China
Gaucher disease
velaglucerase alfa
pharmacokinetics
Online toegang:http://www.sciencedirect.com/science/article/pii/S2214426926000364
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