Fabry Disease
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS trial_result · 2018-09-07Clinical Trial Update: A 24-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease [Phase 2]
A Phase 2 safety and pharmacodynamic study of migalastat hydrochloride (AT1001) in Fabry disease has completed; results were posted in 2018.
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CLINICAL_TRIALS trial_result · 2018-09-07Clinical Trial Update: A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease [Phase 2]
A 12-week Phase 2 safety and pharmacodynamic study of migalastat hydrochloride (AT1001) in participants with Fabry disease has completed wit…
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FDA approval · 2018-08-10FDA Approved: GALAFOLD — Original Application (Amicus Therapeutics US, LLC)
FDA approved GALAFOLD (migalastat hydrochloride) under Original Application from Amicus Therapeutics US, LLC for the treatment of Fabry dise…
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CLINICAL_TRIALS research · 2018-07-23Clinical Trial Update: Epidemiological Study of Fabry Disease in Taiwan Young Stroke Patients
An epidemiological clinical trial update studying Fabry disease among young stroke patients in Taiwan, sponsored by Chiayi Christian Hospita…
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CLINICAL_TRIALS trial_result · 2018-07-10Clinical Trial Update: A Study to Assess the Safety and Tolerability of Lucerastat in Subjects With Fabry Disease [Phase 1]
A Phase 1 trial evaluating the safety and tolerability of lucerastat in Fabry disease has completed.
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CLINICAL_TRIALS withdrawal · 2018-01-08Clinical Trial Update: Study to Evaluate the Safety and EffIcacy of PRX-102 on Gastrointestinal Symptoms in Naïve Fabry Disease [Phase 3]
Phase 3 study evaluating PRX-102 for gastrointestinal symptoms in Fabry disease has been withdrawn.
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CLINICAL_TRIALS trial_result · 2017-12-13Clinical Trial Update: Pharmacokinetics, Pharmacodynamics, and Safety of Moss-aGalactosidase A in Patients With Fabry Disease [Phase 1]
This Phase 1 study reports pharmacokinetics, pharmacodynamics, and safety data for Moss-aGalactosidase A in patients with Fabry disease.
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CLINICAL_TRIALS research · 2017-12-05Clinical Trial Update: Diagnostic Value of the Electrocardiogram in Fabry Disease
A clinical trial update evaluating whether electrocardiography can aid in diagnosing Fabry disease.
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CLINICAL_TRIALS trial_result · 2017-08-28Clinical Trial Update: Podocyturia - Predictor of Renal Dysfunction in Fabry Nephropathy
A clinical trial update examining whether podocyturia can predict renal dysfunction in Fabry disease–related nephropathy; current status is …
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CLINICAL_TRIALS trial_result · 2017-08-03Clinical Trial Update: Safety and Pharmacokinetics of AT1001 (Migalastat HCl) in Healthy Subjects and Subjects With Impaired Renal Function [Phase 1]
Phase 1 safety and pharmacokinetic study of migalastat HCl (AT1001) in healthy subjects and in subjects with impaired renal function, in Fab…
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CLINICAL_TRIALS research · 2017-07-19Clinical Trial Update: Natural History in Fabry Patients With IVS4+919G>A Mutation
This clinical trial update reports on the natural history of Fabry disease in patients with the IVS4+919G>A mutation, with emphasis on cardi…
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CLINICAL_TRIALS trial_result · 2017-07-02Clinical Trial Update: Analysis of the Nervous System in Patients With Fabry's Disease
Completed clinical trial analysis examining nervous system involvement in patients with Fabry disease.
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CLINICAL_TRIALS trial_result · 2017-07-02Clinical Trial Update: Study to Collect Data on Fabry Disease Patients With Enhanceable Alpha-Galactosidase A Activity
This clinical trial update reports the completion of a study collecting data on Fabry disease patients with enhanceable alpha-galactosidase …
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CLINICAL_TRIALS research · 2017-07-02Clinical Trial Update: Registry of Fabry Disease - A Multicenter Observational Study
This update reports that the Registry of Fabry Disease, a multicenter observational study, has been completed.
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CLINICAL_TRIALS research · 2017-06-26Clinical Trial Update: Native T1 Mapping by Cardiovascular Magnetic Resonance Imaging in Rare Diseases
Clinical trial update on native T1 mapping by cardiovascular MRI in rare diseases, including Fabry disease, sponsored by University College …
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CLINICAL_TRIALS research · 2017-05-08Clinical Trial Update: Lipidomics and Functional Analyses of Platelets in Fabry Disease
The clinical trial update studies lipidomics and platelet function in Fabry disease. The study status is unknown.
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CLINICAL_TRIALS research · 2017-04-21Clinical Trial Update: Study of the Relation Between Lipid Myocardial Overload Evaluated by Cardiac Magnetic Resonance Imaging (MRI), Alteration of Longitudinal Myocardial Deformations by Echocardiography, and Clinical Achievements (Functional, Biological and Electrical) in Fabry Disease, and Its Outcomes.
This clinical trial update investigates how lipid-related myocardial overload detected by cardiac MRI relates to longitudinal myocardial def…
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CLINICAL_TRIALS trial_result · 2017-04-04Clinical Trial Update: Immune Response in Subjects With Fabry Disease Who Are Switching From Agalsidase Alfa to Agalsidase Beta
The completed trial evaluated immune responses in Fabry disease patients who switched from agalsidase alfa to agalsidase beta.
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CLINICAL_TRIALS research · 2017-04-04Clinical Trial Update: Investigating Lysosomal Storage Diseases in Minority Groups
A clinical trial update from O & O Alpan LLC examining lysosomal storage disorders in minority populations, including Gaucher disease, Fabry…
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CLINICAL_TRIALS research · 2017-01-13Clinical Trial Update: Establishment of Biomarkers for Fabry Disease
A completed clinical trial update describing the establishment of biomarkers for Fabry disease, aiming to support diagnosis and monitoring.
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FDA approval · 2016-12-20FDA Approved: ELELYSO — Supplemental Application (Pfizer Laboratories Div Pfizer Inc)
FDA approved a supplemental application for ELELYSO (taliglucerase alfa) from Pfizer Laboratories Div Pfizer Inc.
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CLINICAL_TRIALS research · 2016-08-30Clinical Trial Update: Diagnosis of Fabry Disease: Impact of an Educational Brochure Intended for Cardiologist
A clinical trial update evaluating the effect of an educational brochure for cardiologists on identifying Fabry disease, particularly in the…
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CLINICAL_TRIALS research · 2016-08-10Clinical Trial Update: Fabry Disease in Cerebrovascular Disease
This clinical trial update from Chang Gung Memorial Hospital concerns Fabry disease in the context of cerebrovascular disease, and the study…
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CLINICAL_TRIALS research · 2016-07-25Clinical Trial Update: Study of the Prevalence of Fabry Disease in French Dialysis Patients
A clinical trial update describing a study to determine how prevalent Fabry disease is among French patients with end-stage renal disease un…
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CLINICAL_TRIALS trial_result · 2016-06-29Clinical Trial Update: A Study of Two Fabrazyme (Agalsidase Beta) Dosing Regimens in Treatment-naïve, Male Pediatric Patients Without Severe Symptoms [Phase 3]
A Phase 3 trial comparing two dosing regimens of Fabrazyme (agalsidase beta) in treatment-naïve, male pediatric Fabry disease patients has c…