OMIX005209

1Summary
Title A Phase 4, open label, safety and efficacy study of Fabrazyme (agalsidase beta) as enzyme replacement therapy in Chinese participants with Fabry disease
Description Based on the Phase 3 study and its extension study as well as the Phase 4 study results, it was demonstrated that early treatment may alleviate symptoms and prevent further progression of this lethal disease before irreversible damage has occurred.The current study is to evaluate the safety and the efficacy of 1 mg/kg of agalsidase beta every 2 weeks in a Chinese population in the post-marketing setting.
Organism Homo sapiens
Data Type Expression Profiling
Data Accessibility Controlled-access
BioProject PRJCA020590
Release Date 2024-12-31
Submitter Nan Chen (cnrj100@126.com)
Organization Ruijin Hospital, Shanghai jiaotong University School of Medicine
Submission Date 2023-11-07
2Files & Download

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File ID File Title Number/Samples File Type File Size File Suffix Download
OMIX005209-01 PAC-GLA mutation-V1 22 Expression Profiling 11.5 KB xlsx Controlled
3Relevant Publications
Paper Title Journal Name Publish Time Accession Citing Type
A phase 4, open-label, multicenter study of the safety and efficacy of agalsidase beta in Chinese patients with Fabry disease Orphanet Journal of Rare Diseases 2025-08 OMIX005209 Deposit

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