Fabry Disease
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS research · 2026-04-24Clinical Trial Update: A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants [Phase 3]
This is a Phase 3 clinical trial update for migalastat in pediatric subjects aged 2 to under 12 with Fabry disease and amenable GLA variants…
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CLINICAL_TRIALS research · 2026-04-16Clinical Trial Update: Fabry Cardiomyopathy: Identification of Early Myocardial Structural and Tissue Abnormalities Using Multiparametric MRI [NA]
A multicenter clinical trial led by University Health Network in Toronto is evaluating multiparametric MRI to identify early myocardial stru…
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CLINICAL_TRIALS research · 2026-04-03Clinical Trial Update: A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease [Phase 3]
Phase 3 study evaluating migalastat in Fabry disease patients with amenable GLA variants and renal disease; the study is currently active bu…
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CLINICAL_TRIALS research · 2026-04-01Clinical Trial Update: Fabry Disease Registry & Pregnancy Sub-registry
A Fabry disease patient registry and a pregnancy sub-registry sponsored by Genzyme is currently recruiting to collect clinical data on indiv…
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CLINICAL_TRIALS research · 2026-04-01Clinical Trial Update: Natural History in Fabry Disease With IVS4+919G>A Mutations
Observational clinical trial update for a natural history study in Fabry disease patients carrying the IVS4+919G>A mutation; the trial is sp…
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CLINICAL_TRIALS research · 2026-03-27Clinical Trial Update: Anderson-Fabry Disease Fitness Improvement Training: A-FAD-FIT [NA]
The study A-FAD-FIT is currently enrolling by invitation to assess fitness improvement training in individuals with Fabry disease.
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CLINICAL_TRIALS research · 2026-03-24Clinical Trial Update: T1 Mapping in Fabry Disease [NA]
This clinical trial update reports that a study on T1 mapping in Fabry disease is currently recruiting.
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CLINICAL_TRIALS research · 2026-03-20Clinical Trial Update: Care Pathway for Patients With Fabry's Disease (Fabry-PATH)
This entry describes a clinical trial protocol named Fabry-PATH to develop a care pathway for Fabry disease. The study is not yet recruiting…
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CLINICAL_TRIALS research · 2026-03-19Clinical Trial Update: A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease [Phase 2, Phase 3]
Ongoing Phase 2/3 clinical trial evaluating PRX-102 in children and adolescents with Fabry disease, currently recruiting.
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CLINICAL_TRIALS research · 2026-03-19Clinical Trial Update: Maternal and Postnatal Outcomes Study (MOS): A Global Observational Registry Assessing the Safety of Elfabrio® in Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
Global observational registry MOS investigates the safety of Elfabrio (migalastat) in pregnant women with Fabry disease and their infants du…
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CLINICAL_TRIALS research · 2026-03-18Clinical Trial Update: Study to Evaluate the Safety, PK, PD, and Efficacy of PRX-102 in Japanese Patients With Fabry Disease [Phase 2, Phase 3]
A Phase 2/Phase 3 clinical trial is evaluating the safety, pharmacokinetics, pharmacodynamics, and efficacy of PRX-102 in Japanese patients …
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CLINICAL_TRIALS research · 2026-03-13Clinical Trial Update: A Study of Patients With Fabry Disease (US Specific)
A US-specific Fabry disease clinical trial sponsored by Amicus Therapeutics is currently recruiting participants.
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CLINICAL_TRIALS research · 2026-02-27Clinical Trial Update: Evaluate the Safety and Preliminary Efficacy of EXG110 in Subjects With Fabry Disease [NA]
This document reports a clinical trial evaluating the safety and preliminary efficacy of EXG110 in Fabry disease, with the study currently r…
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CLINICAL_TRIALS research · 2026-02-24Clinical Trial Update: Observational Study on Long-term Use of Pegunigalsidase Alfa in Fabry Patients in a Real-world Setting
An observational clinical trial to assess long-term use of pegunigalsidase alfa in Fabry disease patients in real-world settings; the study …
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CLINICAL_TRIALS research · 2026-02-24Clinical Trial Update: RAre, But Not aLone: a Large Italian Network to Empower the Impervious diaGNostic Pathway of Rare cerEbrovascular Diseases (ALIGNED)
A large Italian network study called ALIGNED aims to improve diagnostic pathways for rare cerebrovascular diseases and is currently recruiti…
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CLINICAL_TRIALS research · 2026-02-18Clinical Trial Update: Long Duration Holter ECG in Fabry Disease [NA]
A clinical trial sponsored by INSERM is evaluating long-duration Holter ECG monitoring in Fabry disease and is currently recruiting in Franc…
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CLINICAL_TRIALS research · 2026-02-04Clinical Trial Update: A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease [Phase 3]
A Phase 3 clinical trial evaluating Venglustat tablets for neuropathic and abdominal pain in people with Fabry disease is currently active b…
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CLINICAL_TRIALS research · 2026-01-21Clinical Trial Update: Precision Diagnosis and Risk Stratification of Rare Cardiomyopathies Based on Novel Cardiac Magnetic Resonance Techniques
Clinical trial evaluating precision diagnosis and risk stratification of rare cardiomyopathies using novel cardiac MRI techniques. The study…
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CLINICAL_TRIALS research · 2026-01-20Clinical Trial Update: Novel Diagnostic and Prognostic Predictors in Fabry Cardiomyopathy: Proof of Concept in a Rare Disease
This clinical trial update outlines a proof-of-concept study investigating novel diagnostic and prognostic predictors for Fabry cardiomyopat…
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CLINICAL_TRIALS research · 2026-01-20Clinical Trial Update: Taiwan Associated Genetic and Nongenetic Small Vessel Disease
This Taiwan-based clinical trial is recruiting to study genetic and non-genetic cerebral small vessel disease, including CADASIL, HTRA1-rela…
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CLINICAL_TRIALS research · 2026-01-02Clinical Trial Update: A Study of Agalsidase Alfa Enyzme Replacement Therapy in Chinese Children and Adults With Fabry Disease
This ongoing clinical trial in Chinese children and adults with Fabry disease is evaluating enzyme replacement therapy with agalsidase alfa.…
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CLINICAL_TRIALS research · 2025-12-31Clinical Trial Update: Efficacy and Safety of Enzyme Replacement Therapy in Patients With Fabry Disease
This update describes an ongoing clinical trial evaluating the efficacy and safety of enzyme replacement therapy in patients with Fabry dise…
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CLINICAL_TRIALS research · 2025-12-11Clinical Trial Update: Study of the Quality of Life of Patients With Fabry Disease Aged 65 and Over With and Without Specific Treatment
This ClinicalTrials.gov entry reports on a study evaluating the quality of life of Fabry disease patients aged 65 and older, comparing outco…
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CLINICAL_TRIALS research · 2025-12-01Clinical Trial Update: Effect of Agalsidase Alfa on Cardiac Inflammation in Patients With Fabry Disease: A [18F]-FDG PET-CMR Study
A clinical trial is evaluating the effect of agalsidase alfa on cardiac inflammation in Fabry disease patients using 18F-FDG PET-CMR imaging…
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CLINICAL_TRIALS research · 2025-11-14Clinical Trial Update: A Multi-Country Observational Study of Safety and Effectiveness of Elfabrio® in Fabry Patients
A multi-country observational study evaluating the safety and effectiveness of Elfabrio (migalastat) in Fabry disease patients, sponsored by…