Fabry Disease
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS trial_result · 2019-02-15Clinical Trial Update: Fabry's Disease and Pregnancy (PREFAB)
The PREFAB clinical trial investigating Fabry disease and pregnancy has been terminated.
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CLINICAL_TRIALS trial_result · 2018-11-01Clinical Trial Update: Drug-Drug Interaction Study Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Participants With Fabry Disease [Phase 2]
Phase 2 study evaluating drug-drug interaction between migalastat hydrochloride (AT1001) and agalsidase in Fabry disease completed with post…
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CLINICAL_TRIALS trial_result · 2018-11-01Clinical Trial Update: Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease [Phase 3]
A Phase 3 trial comparing oral AT1001 with enzyme replacement therapy in Fabry disease has completed with results posted in 2018.
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CLINICAL_TRIALS trial_result · 2018-10-30Clinical Trial Update: Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease [Phase 3]
Phase 3 trial assessing the effects of oral migalastat hydrochloride (AT1001) in Fabry disease has completed with results posted.
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CLINICAL_TRIALS trial_result · 2018-10-03Clinical Trial Update: Open-label Long-term Safety Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease Who Have Completed a Previous AT1001 Study [Phase 2]
Open-label long-term safety study of migalastat hydrochloride (AT1001) in Fabry disease participants who completed a prior AT1001 study; ter…
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CLINICAL_TRIALS trial_result · 2018-10-02Clinical Trial Update: Open-Label Phase 3 Long-Term Safety Study of Migalastat [Phase 3]
Open-label Phase 3 long-term safety study of migalastat in Fabry disease was terminated; results posted in 2018.
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CLINICAL_TRIALS trial_result · 2018-09-07Clinical Trial Update: A Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease [Phase 2]
Phase 2 clinical trial update for migalastat hydrochloride (AT1001) in Fabry disease completed with posted results.
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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 Female Participants With Fabry Disease [Phase 2]
Phase 2 safety and pharmacodynamic study of migalastat hydrochloride (AT1001) in female participants with Fabry disease completed.
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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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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 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 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 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 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 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…
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CLINICAL_TRIALS trial_result · 2016-06-24Clinical Trial Update: Evaluate the Safety and Exploratory Efficacy of GC1119 [Phase 1]
A Phase 1 clinical trial evaluating GC1119 for Fabry disease has completed. The study assessed safety and exploratory efficacy of GC1119.
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CLINICAL_TRIALS trial_result · 2016-01-13Clinical Trial Update: Androgenetic Alopecia in Fabry Disease
This document reports the completion of a clinical trial investigating androgenetic alopecia in patients with Fabry disease.
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CLINICAL_TRIALS trial_result · 2015-05-08Clinical Trial Update: A Long Term Safety and Efficacy Study of Fabrazyme Replacement Therapy in Japanese Patients With Fabry Disease.
This clinical trial update describes the long-term safety and efficacy study of Fabrazyme (agalsidase beta) in Japanese patients with Fabry …
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CLINICAL_TRIALS trial_result · 2015-04-15Clinical Trial Update: A Safety and Efficacy Study of Fabrazyme® Replacement Therapy in Patients With Cardiac Fabry Disease [Phase 4]
Phase 4 study evaluating the safety and efficacy of Fabrazyme replacement therapy in patients with cardiac Fabry disease. Results have been …
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CLINICAL_TRIALS trial_result · 2015-03-31Clinical Trial Update: Dosing Study of Replagal in Patients With Fabry Disease [Phase 2]
This clinical trial update reports a Phase 2 dosing study of Replagal (agalsidase alfa) in patients with Fabry disease, with results posted …
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CLINICAL_TRIALS trial_result · 2014-11-10Clinical Trial Update: Enzyme Replacement Therapy in Fabry Disease
The clinical trial NCT00149318 evaluating enzyme replacement therapy for Fabry disease sponsored by the Mario Negri Institute has been termi…
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CLINICAL_TRIALS trial_result · 2014-10-28Clinical Trial Update: Ophthalmic Findings During 10-year Enzyme Substitution of Danish Fabry Patients. [Phase 4]
This Phase 4 update summarizes ophthalmic findings from 10 years of enzyme replacement therapy in Danish Fabry disease patients.