A phase 4, open-label, multicenter study of the safety and efficacy of agalsidase beta in Chinese patients with Fabry disease

Abstract Background This is the first phase 4 study evaluating safety and efficacy of enzyme replacement therapy (ERT) in Chinese patients with Fabry disease, and exploring the impact of COVID-19 infection on the prognosis of Fabry disease under ERT. Methods and results Eligible patients received an...

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Bibliographic Details
Main Authors: Hong Ren, Wei Zhang, Yan Ouyang, Junhong Guo, Hong Xu, Jie Ma, Xiaoping Luo, Xiaoxia Pan, Yun Yuan, Qian Shen, Bin Li, Qiqi Feng, Shi Liu, Nan Chen
Format: Article
Language:English
Published: BMC 2025-08-01
Series:Orphanet Journal of Rare Diseases
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Online Access:https://doi.org/10.1186/s13023-025-03950-7
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