Fabry Disease
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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 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 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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EMA approval · 2025-12-19EMA Authorised: Replagal (agalsidase alfa) — Takeda Pharmaceuticals International AG Ireland Branch
The EMA authorised Replagal (agalsidase alfa) for treatment of Fabry disease, with Takeda Pharmaceuticals International AG Ireland Branch as…
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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…
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CLINICAL_TRIALS research · 2025-11-04Clinical Trial Update: A proof-of Concept Study to Assess Safety and Tolerability of HM15421/GC1134A in Patients With Fabry Disease [Phase 1, Phase 2]
A phase 1/2 proof-of-concept trial is assessing safety and tolerability of HM15421/GC1134A in patients with Fabry disease; study is currentl…
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CLINICAL_TRIALS research · 2025-10-23Clinical Trial Update: Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease [Phase 1, Phase 2]
This is a clinical trial update describing a Phase 1/2 study of AMT-191 in classic Fabry disease, assessing safety, PK/PD, and exploratory e…
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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 research · 2025-09-12Clinical Trial Update: ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
This clinical trial update describes ScreenPlus, a comprehensive, multi-disorder newborn screening program that is currently enrolling by in…
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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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EMA approval · 2025-08-25EMA Authorised: Galafold (migalastat hydrochloride) — Amicus Therapeutics Europe Limited
The EMA authorised Galafold (migalastat hydrochloride) by Amicus Therapeutics Europe Limited for Fabry disease, with orphan medicine designa…
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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 research · 2025-08-22Clinical Trial Update: Natural History and Structural Functional Relationships in Fabry Renal Disease Treatment Outcomes(Changes)in Fabry Renal Disease Study
ClinicalTrials.gov listing for a study on the natural history and structural-functional relationships in Fabry disease with renal involvemen…
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CLINICAL_TRIALS research · 2025-07-08Clinical Trial Update: A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
A global prospective observational study sponsored by Amicus Therapeutics, evaluating pregnancy and breastfeeding outcomes in women with Fab…
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CLINICAL_TRIALS research · 2025-07-03Clinical Trial Update: Physician Initiated Expanded Access Request for Migalastat in Individual Patients With Fabry Disease
A physician-initiated expanded access request for migalastat in individual Fabry disease patients is being tracked, with the program listed …