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 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 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 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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EMA approval · 2026-03-31EMA Authorised: Elfabrio (pegunigalsidase alfa) — Chiesi Farmaceutici S.p.A
EMA authorised Elfabrio (pegunigalsidase alfa) from Chiesi Farmaceutici S.p.A for Fabry disease.
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CLINICAL_TRIALS other · 2026-03-27Clinical Trial Update: A Study of Home vs Hospital Treatment in People With Fabry, Gaucher or Hunter Conditions in Mexico
ClinicalTrials.gov update on a study comparing home vs hospital treatment for Fabry disease, Gaucher disease, and Hunter syndrome in Mexico,…
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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 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 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 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 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 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 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 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…