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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Λεπτομέρειες βιβλιογραφικής εγγραφής

Κύριοι συγγραφείς:	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
Μορφή:	Artigo
Γλώσσα:	Inglês
Έκδοση:	Elsevier 2026-06-01
Σειρά:	Molecular Genetics and Metabolism Reports
Θέματα:	China Gaucher disease velaglucerase alfa pharmacokinetics
Διαθέσιμο Online:	http://www.sciencedirect.com/science/article/pii/S2214426926000364
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