Fabry Disease
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS trial_result · 2023-02-08Clinical Trial Update: Extension Study of PRX-102 for up to 60 Months [Phase 1, Phase 2]
Extension study update for PRX-102 in Fabry disease following Phase 1/2, with results posted after completion.
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CLINICAL_TRIALS trial_result · 2023-02-02Clinical Trial Update: Effects Of Sodium Glucose Cotranspoter 2 Inhibitors On Heart And Kidneys In Fabry Disease Patients [Phase 2]
Phase 2 trial update investigating SGLT2 inhibitors for effects on heart and kidneys in Fabry disease patients.
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CLINICAL_TRIALS trial_result · 2023-01-05Clinical Trial Update: Safety, Efficacy, & PK of PRX-102 in Patients With Fabry Disease Administered Intravenously Every 4 Weeks [Phase 3]
Phase 3 trial assessing safety, efficacy, and pharmacokinetics of PRX-102 given IV every 4 weeks in Fabry disease completed with posted resu…
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CLINICAL_TRIALS trial_result · 2022-06-29Clinical Trial Update: Safety and Efficacy of PRX-102 in Patients With Fabry Disease Currently Treated With REPLAGAL® (Agalsidase Alfa) [Phase 3]
Phase 3 trial updating safety and efficacy of PRX-102 in Fabry disease patients already treated with agalsidase alfa (REPLAGAL).
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FDA approval · 2021-12-10FDA Approved: GALAFOLD — Supplemental Application (Amicus Therapeutics US, LLC)
The FDA approved a supplemental application for GALAFOLD (migalastat hydrochloride) to expand its labeling for Fabry disease.
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CLINICAL_TRIALS trial_result · 2021-11-30Clinical Trial Update: Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Years) [Phase 3]
Phase 3 pediatric study assessing migalastat safety, pharmacokinetics/pharmacodynamics, and efficacy in adolescents with Fabry disease has c…
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CLINICAL_TRIALS trial_result · 2021-10-07Clinical Trial Update: Assessment of Small Fiber Neuropathy in Rare Diseases Using Sudoscan [NA]
A completed clinical trial assessing small fiber neuropathy in Fabry disease and related conditions using Sudoscan; results posted in 2021.
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FDA approval · 2021-03-11FDA Approved: FABRAZYME — Supplemental Application (Genzyme Corporation)
FDA approved a supplemental Biologics License Application for FABRAZYME (agalsidase beta) from Genzyme Corporation.
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CLINICAL_TRIALS trial_result · 2020-12-21Clinical Trial Update: Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease [Phase 3]
Phase 3 open-label extension study reporting long-term effects of migalastat HCL in Fabry disease has completed with results posted.
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FDA label_change · 2020-09-25FDA Approved: GALAFOLD — Supplemental Application (Amicus Therapeutics US, LLC)
The FDA approved a supplemental application for GALAFOLD (migalastat hydrochloride) submitted by Amicus Therapeutics US, LLC to update its l…
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CLINICAL_TRIALS trial_result · 2020-01-27Clinical Trial Update: Dose-ranging Study of PRX-102 in Adult Fabry Disease Patients [Phase 1, Phase 2]
Dose-ranging study of PRX-102 in adult Fabry disease patients completed; results posted in 2020.
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CLINICAL_TRIALS trial_result · 2019-12-17Clinical Trial Update: Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-naïve Adult Male Patients With Fabry Disease [Phase 2]
Phase 2 clinical trial evaluating safety, pharmacodynamics, pharmacokinetics, and exploratory efficacy of GZ/SAR402671 in treatment-naïve ad…
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CLINICAL_TRIALS trial_result · 2019-12-09Clinical Trial Update: Evaluation of the Long-term Safety, Pharmacodynamics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-Naïve Adult Male Patients With Fabry Disease [Phase 2]
Phase 2 trial evaluating long-term safety, pharmacodynamics, and exploratory efficacy of GZ/SAR402671 in treatment-naïve adult male Fabry di…
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FDA approval · 2019-11-01FDA Approved: FABRAZYME — Supplemental Application (Genzyme Corporation)
The FDA approved a supplemental application for Fabrazyme (agalsidase beta) from Genzyme Corporation.
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FDA label_change · 2019-08-22FDA Approved: GALAFOLD — Supplemental Application (Amicus Therapeutics US, LLC)
FDA approved a supplemental application for GALAFOLD (migalastat hydrochloride) by Amicus Therapeutics US, LLC, resulting in labeling update…
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CLINICAL_TRIALS trial_result · 2019-02-15Clinical Trial Update: A Multicenter Open-Label Treatment Protocol to Observe the Safety of Replagal (Agalsidase Alfa) Enzyme Replacement Therapy in Canadian Patients With Fabry Disease [Phase 3]
A multicenter open-label trial assessing the safety of agalsidase alfa enzyme replacement therapy in Canadian Fabry disease patients; study …
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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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FDA approval · 2018-12-18FDA Approved: FABRAZYME — Supplemental Application (Genzyme Corporation)
The FDA approved a supplemental application for FABRAZYME (agalsidase beta) from Genzyme Corporation.
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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.