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Agalsidase Beta Long-Term Treatment Outcome for Fabry Disease Patients With IVS4 Mutation in Taiwan
Sponsor: Sanofi
Summary
This is a national, multicenter, observational, cohort study designed to assess clinical outcomes upon agalsidase beta treatment, to characterize the clinical manifestations, and to collect the natural history on male and female Fabry disease adult patients who carry the GLA IVS4. This study aims to retrospectively and prospectively investigate the disease natural history, clinical manifestations, and the treatment outcomes upon agalsidase beta in Fabry disease (FD) patients carrying the GLA IVS4 mutation from medical records, physician assessments, and patient-reported outcomes.
Official title: An Observational Study of Enzyme Replacement Therapy-Naïve and Agalsidase Beta-Treated Fabry Disease Patients With GLA IVS4 919 G>A Mutation in Taiwan
Key Details
Gender
All
Age Range
18 Years - Any
Study Type
OBSERVATIONAL
Enrollment
78
Start Date
2023-09-13
Completion Date
2026-09-10
Last Updated
2025-03-21
Healthy Volunteers
No
Conditions
Locations (5)
Investigational Site Number : 1580004
Taichung, Taiwan
Investigational Site Number : 1580005
Tainan, Taiwan
Investigational Site Number : 1580001
Taipei, Taiwan
Investigational Site Number : 1580003
Taipei, Taiwan
Investigational Site Number : 1580002
Taipei, Taiwan