Fabry Disease
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Sign up freeTop drugs studied for this disease
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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 research · 2025-05-14Clinical Trial Update: Arrhythmia Burden, Risk of Sudden Cardiac Death and Stroke in Patients With Fabry Disease [NA]
Clinical trial update on arrhythmia burden and risk of sudden cardiac death and stroke in patients with Fabry disease; the study is currentl…
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CLINICAL_TRIALS research · 2025-05-04Clinical Trial Update: Aortic Dimensions in Patients With Fabry Disease
A clinical trial evaluating aortic dimensions in patients with Fabry disease is currently not yet recruiting, sponsored by University Hospit…
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CLINICAL_TRIALS research · 2025-04-08Clinical Trial Update: the Role of cArdiac Inflammation, endoThelial Dysfunction, and FIbrosis in fabrY Disease
This clinical trial investigates the role of cardiac inflammation, endothelial dysfunction, and fibrosis in Fabry disease and is currently r…
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CLINICAL_TRIALS research · 2025-04-06Clinical Trial Update: Fibrosis, Inflammation, Oxygenation of Renal Tissue In FabrY Disease
A clinical trial is recruiting to study fibrosis, inflammation, and oxygenation in renal tissue for Fabry disease with chronic kidney diseas…
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CLINICAL_TRIALS research · 2025-03-30Clinical Trial Update: German Observational Multicenter Study of Patients With Fabry Disease Under Enzyme Replacement Therapy With Pegunigalsidase-alfa
This clinical trial update describes an observational multicenter study in Germany evaluating patients with Fabry disease who are receiving …
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CLINICAL_TRIALS research · 2025-03-26Clinical Trial Update: CVI Alterations in FD: a Prospective, Multicenter, Observational Cohort Study
A prospective, multicenter observational cohort study evaluating cardiovascular alterations in Fabry disease with cardiac involvement.
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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 research · 2025-02-21Clinical Trial Update: 4D-310 in Adults With Fabry Disease and Cardiac Involvement [Phase 1, Phase 2]
An ongoing Phase 1/2 clinical trial evaluating 4D-310 in adults with Fabry disease and cardiac involvement is currently recruiting.
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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 research · 2025-02-11Clinical Trial Update: The Safety and Efficacy of Intravenous EXG110 in Patients With Fabry Disease [Phase 1, Phase 2]
This document reports a Phase 1/2 clinical trial evaluating intravenous EXG110 in patients with Fabry disease, conducted by Guangzhou Jiayin…
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CLINICAL_TRIALS research · 2025-02-07Clinical Trial Update: Characterization of Patients With Cardiomyopathy to Identify Critical Patients Candidates for Cardiac Transplantation
This clinical trial update describes a study at IRCCS Azienda Ospedaliero-Universitaria di Bologna that aims to characterize patients with v…
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CLINICAL_TRIALS research · 2025-02-04Clinical Trial Update: Italian Anderson Fabry Disease Cardiovascular Registry
This clinical trial registry entry describes the Italian Anderson Fabry Disease Cardiovascular Registry, a recruiting project to collect car…
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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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FDA approval · 2025-01-17FDA Approved: ELELYSO — Supplemental Application (Pfizer Laboratories Div Pfizer Inc)
The FDA approved a supplemental application for ELELYSO (taliglucerase alfa) submitted by Pfizer Laboratories Div Pfizer Inc.
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CLINICAL_TRIALS research · 2025-01-06Clinical Trial Update: Characterizing the Retinal Microvasculature in Patients with Fabry Disease: a Prospective Observational Study
A prospective observational study at Technical University of Munich aims to characterize the retinal microvasculature in Fabry disease patie…
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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…
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CLINICAL_TRIALS trial_result · 2024-10-01Clinical Trial Update: Studying Lipids as Potential Biomarkers in Patients With Fabry Disease
A completed clinical trial update studying lipids as potential biomarkers in patients with Fabry disease.
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CLINICAL_TRIALS trial_result · 2024-09-19Clinical Trial Update: Clinical Investigation on the Blood Oxygenation at the Optic Nerve Head in Fabry Patients
This entry reports the completion and posting of results from a clinical investigation examining blood oxygenation at the optic nerve head i…
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EMA approval · 2024-09-04EMA Authorised: Fabrazyme (agalsidase beta) — Sanofi B.V.
The European Medicines Agency authorised Fabrazyme (agalsidase beta) for Fabry disease, with Sanofi B.V. as the marketing authorization hold…