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

Authors

Hong Ren (ORCID: https://orcid.org/0000-0002-4557-0918)
Wei Zhang (ORCID: https://orcid.org/0000-0002-5822-9970)
Xiaoping Luo (ORCID: https://orcid.org/0000-0002-3748-4817)
Jie Ma (ORCID: https://orcid.org/0000-0002-8934-922X)
Shi Liu (ORCID: https://orcid.org/0000-0003-2289-9160)
Yun Yuan (ORCID: https://orcid.org/0000-0003-4736-199X)
Bin Li (ORCID: https://orcid.org/0000-0002-8442-0154)
Junhong Guo (ORCID: https://orcid.org/0009-0008-4724-7138)
Nan Chen
Qiqi Feng
Qian Shen (ORCID: https://orcid.org/0000-0003-4210-3131)
Yan Ouyang (ORCID: https://orcid.org/0000-0002-3181-8708)
Xiaoxia Pan (ORCID: https://orcid.org/0000-0002-2963-7019)
Hong Xu (ORCID: https://orcid.org/0009-0005-3027-8104)

Institutions

Shanxi Medical University (CN)
Chinese Academy of Medical Sciences & Peking Union Medical College (CN)
Peking University (CN)
Peking Union Medical College Hospital (CN)
Ruijin Hospital (CN)
First Hospital of Shanxi Medical University (CN)
Peking University First Hospital (CN)
Sanofi (China) (CN)
Children's Hospital of Fudan University (CN)
Tongji Hospital (CN)
Union Hospital (CN)
Huazhong University of Science and Technology (CN)

Publication Details

Journal: Orphanet Journal of Rare Diseases
Published: 2025-08-04
DOI: https://doi.org/10.1186/s13023-025-03950-7
Primary Topic: Lysosomal Storage Disorders Research
Type: article
Field-Weighted Citation Impact: 0.00

Funders

Sanofi
Sanofi China Investment Company

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article

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

Hong Ren, Wei Zhang, Xiaoping Luo, Jie Ma et al.

Aug 4, 2025

Orphanet Journal of Rare Diseases

Lysosomal Storage Disorders Research

article

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

Hong Ren, Wei Zhang, Xiaoping Luo, Jie Ma, Shi Liu, Yun Yuan, Bin Li, Junhong Guo, Nan Chen, Qiqi Feng, Qian Shen, Yan Ouyang, Xiaoxia Pan, Hong Xu

August 4, 2025article en

Abstract

No abstract available for this paper.

Orphanet Journal of Rare DiseasesVol. 20(1)

Shanxi Medical University (CN), Chinese Academy of Medical Sciences & Peking Union Medical College (CN), Peking University (CN), Peking Union Medical College Hospital (CN), Ruijin Hospital (CN), First Hospital of Shanxi Medical University (CN), Peking University First Hospital (CN), Sanofi (China) (CN), Children's Hospital of Fudan University (CN), Tongji Hospital (CN), Union Hospital (CN), Huazhong University of Science and Technology (CN)

Sanofi, Sanofi China Investment Company

Good health and well-being

Openalex Percentile: Top 75%

Lysosomal Storage Disorders Research

DOI Open Access PDF OpenAlex

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