Fabry Disease
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS trial_result · 2026-08-06Clinical Trial Results: A Fabry Disease Gene Therapy Study [Phase 1, Phase 2]
Phase 1/2 trial results for a Fabry disease gene therapy were posted after termination of the study.
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CLINICAL_TRIALS trial_result · 2026-04-14Clinical Trial Update: Dose-Ranging Study of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease (STAAR) [Phase 1, Phase 2]
This clinical trial update reports the completion of a Phase 1/2 dose-ranging study of ST-920, an AAV2/6-based gene therapy delivering alpha…
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CLINICAL_TRIALS trial_result · 2026-03-24Clinical Trial Update: Open Label Extension Study of 1 mg/kg Pegunigalsidase Alfa Every 2 Weeks in Patients With Fabry Disease [Phase 3]
This open-label extension of pegunigalsidase alfa at 1 mg/kg every 2 weeks in Fabry disease has completed Phase 3, and results were posted o…
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CLINICAL_TRIALS trial_result · 2026-03-16Clinical Trial Update: A Real-world Wearables Study in Fabry Disease.
A real-world wearable study in Fabry disease has completed, providing final observational data collected via wearables.
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CLINICAL_TRIALS trial_result · 2026-03-16Clinical Trial Update: Early Detection of Fabry Disease
Takeda-sponsored clinical trial evaluating early detection of Fabry disease has completed.
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CLINICAL_TRIALS trial_result · 2026-03-10Clinical Trial Update: A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease [Phase 3]
Phase 3 clinical trial evaluating the effect of Venglustat tablets on left ventricular mass index in adults with Fabry disease. The study is…
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CLINICAL_TRIALS trial_result · 2026-03-04Clinical Trial Update: A One Visit Follow Up of Adults With Fabry Disease Who Started Long-term Enzyme Replacement Therapy As Children [NA]
A completed follow-up study in adults with Fabry disease who began enzyme replacement therapy in childhood to assess long-term outcomes.
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CLINICAL_TRIALS trial_result · 2026-02-18Clinical Trial Update: Switch Over Study of Biosimilar Agalsidase Beta for Fabry Disease [Phase 3]
This clinical trial update reports the completion of a Phase 3 switch-over study of a biosimilar agalsidase beta for Fabry disease, with res…
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CLINICAL_TRIALS trial_result · 2026-01-21Clinical Trial Update: Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients [Phase 3]
Open-label extension of pegunigalsidase alfa (PRX-102) at 2 mg/kg every 4 weeks in adults with Fabry disease; this Phase 3 study is currentl…
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CLINICAL_TRIALS trial_result · 2025-10-21Clinical Trial Update: A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease [Phase 3]
This document provides an update on a Phase 3 long-term safety and tolerability study of Lucerastat in adult patients with Fabry disease.
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CLINICAL_TRIALS trial_result · 2025-10-21Clinical Trial Update: A Prospective Study to Investigate Safety and Tolerability of Shorter Infusion of Fabrazyme [Phase 4]
Phase 4 prospective study evaluating the safety and tolerability of a shorter infusion of Fabrazyme for Fabry disease; results have been pos…
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CLINICAL_TRIALS trial_result · 2025-10-02Clinical Trial Update: Screening of Fabry Disease in Portuguese Patients With Idiopathic Cardiomyopathies
A completed clinical trial evaluating screening for Fabry disease in Portuguese patients with idiopathic cardiomyopathy to assess prevalence…
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CLINICAL_TRIALS trial_result · 2025-09-19Clinical Trial Update: A Study of Replagal in Children and Adults With Fabry Disease in India [Phase 4]
This entry reports a Phase 4 clinical trial evaluating Replagal (agalsidase alfa) in children and adults with Fabry disease in India, curren…
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CLINICAL_TRIALS trial_result · 2025-09-11Clinical Trial Update: China Post-marketing Surveillance (PMS) Study of Fabrazyme® [Phase 4]
A Phase 4 post-marketing surveillance study of Fabrazyme (agalsidase beta) for Fabry disease in China has completed.
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CLINICAL_TRIALS trial_result · 2025-08-22Clinical Trial Update: Anderson Fabry Disease - the Power of Information. [NA]
The update notes completion of a Fabry disease clinical trial sponsored by Fondazione Policlinico Universitario Agostino Gemelli IRCCS. It p…
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CLINICAL_TRIALS trial_result · 2025-06-19Clinical Trial Update: Cardiovasculorenal Phenotyping in Fabry Disease Through Noninvasive Testing
This clinical trial update reports termination of a study on Cardiovasculorenal phenotyping in Fabry disease using noninvasive testing.
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CLINICAL_TRIALS trial_result · 2025-06-17Clinical Trial Update: Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease [Phase 3]
A Phase 3 trial evaluating migalastat in pediatric patients over 12 years with Fabry disease has completed, reporting safety, pharmacodynami…
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CLINICAL_TRIALS trial_result · 2025-05-25Clinical Trial Update: Long-Term Follow-up of Subjects Who Were Treated With ST-920
This update provides long-term follow-up information for subjects treated with ST-920 in Fabry disease.
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CLINICAL_TRIALS trial_result · 2025-05-16Clinical Trial Update: Screening for Fabry Disease in Renal Transplantation
This clinical trial screened renal transplant recipients for Fabry disease; the study (NCT03886714) conducted by University Hospital, Montpe…
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CLINICAL_TRIALS trial_result · 2025-03-21Clinical Trial Update: Agalsidase Beta Long-Term Treatment Outcome for Fabry Disease Patients With IVS4 Mutation in Taiwan
This clinical trial update presents long-term outcomes of agalsidase beta treatment in Fabry disease patients with the IVS4 mutation in Taiw…
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CLINICAL_TRIALS trial_result · 2025-03-17Clinical Trial Update: Autoimmune and Inflammatory Response Biomarkers in Fabry Disease
A completed clinical trial evaluated autoimmune and inflammatory biomarkers in Fabry disease. The study was conducted by Fundacion para la I…
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CLINICAL_TRIALS trial_result · 2025-02-13Clinical Trial Update: A Co-designed Physical Activity Intervention in Fabry Disease
This clinical trial update notes that a co-designed physical activity intervention study in Fabry disease has completed.
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CLINICAL_TRIALS trial_result · 2025-01-30Clinical Trial Update: Diagnostic Role of Renal Biopsy in Patients With Fabry Disease
This completed clinical trial evaluated the diagnostic value of renal biopsy for Fabry disease and its renal manifestations.
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CLINICAL_TRIALS trial_result · 2024-11-04Clinical Trial Update: A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy.
A completed study describes patients' and clinicians' experiences with enzyme replacement therapy for Fabry disease, with results posted.
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CLINICAL_TRIALS trial_result · 2024-10-08Clinical Trial Update: A Study of REPLAGAL® in Treatment-naive Chinese Participants With Fabry Disease [Phase 3]
A Phase 3 study evaluating REPLAGAL (agalsidase alfa) in treatment-naive Chinese participants with Fabry disease has completed and posted re…