Fabry Disease
📧 Sign up to get email alerts when something new happens for Fabry Disease.
Sign up freeTop drugs studied for this disease
-
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.
-
EMA approval · 2016-04-01First oral treatment for Fabry disease recommended for approval in the EU
The EMA recommended approval for the first oral treatment for Fabry disease in the EU, marking a potential new oral option for patients.
-
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.
-
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 …
-
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 …
- Want full summaries, importance scores, and drug timelines? Upgrade to Plus or Pro.
-
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 …
-
CLINICAL_TRIALS research · 2015-03-03Clinical Trial Update: Sleepiness and Sleep-disordered Breathing in Fabry Disease. A Prospective Cohort Study. [NA]
A prospective cohort study evaluating sleepiness and sleep-disordered breathing in Fabry disease, sponsored by the University of Zurich; the…
-
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…
-
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.
-
CLINICAL_TRIALS trial_result · 2014-08-22Clinical Trial Update: Extension Study of TKT028 Evaluating Safety and Clinical Outcomes of Replagal® in Adult Patients With Fabry Disease [Phase 3]
Phase 3 extension study update on Replagal in adults with Fabry disease assessing safety and clinical outcomes.
-
CLINICAL_TRIALS trial_result · 2014-07-10Clinical Trial Update: A Study Evaluating Glycosphingolipid Clearance in Patients Treated With Agalsidase Alfa Who Switch to Agalsidase Beta [Phase 4]
Phase 4 study evaluating glycosphingolipid clearance in Fabry disease patients who switch from agalsidase alfa to agalsidase beta.
-
CLINICAL_TRIALS trial_result · 2014-06-18Clinical Trial Update: A Phase 1 Study To Evaluate the Safety of Migalastat Hydrochloride Given Intravenously to Healthy Volunteers [Phase 1]
A Phase 1 study evaluating the safety of intravenous migalastat hydrochloride in healthy volunteers has completed.
-
CLINICAL_TRIALS research · 2014-06-12Clinical Trial Update: Screening for Fabry Disease Among Young Stroke Patients in an Israeli Stroke Clinic [NA]
Clinical trial update on screening for Fabry disease among young stroke patients at an Israeli stroke clinic. Sponsor: Wolfson Medical Cente…
-
CLINICAL_TRIALS trial_result · 2014-06-06Clinical Trial Update: Safety and Efficacy Study of Several Replagal Dosing Regimens on Cardiac Function in Adults With Fabry Disease [Phase 3]
Phase 3 trial assessing safety and efficacy of several Replagal dosing regimens on cardiac function in adults with Fabry disease completed w…
-
CLINICAL_TRIALS trial_result · 2014-05-28Clinical Trial Update: Pulmonary Involvement in Patients With Fabry Disease
Clinical trial update summarizing a study that investigates pulmonary involvement in Fabry disease, with the study status noted as completed…
-
CLINICAL_TRIALS trial_result · 2014-05-20Clinical Trial Update: Safety Study of Replagal® Therapy in Children With Fabry Disease [Phase 2]
Phase 2 safety study of Replagal therapy in children with Fabry disease has completed with results posted in 2014.
-
CLINICAL_TRIALS trial_result · 2014-04-25Clinical Trial Update: This Study is Designed to Evaluate PD/PK and Safety of Replagal Manufactured by Two Different Processes. [Phase 2]
Phase 2 clinical trial evaluating pharmacokinetics, pharmacodynamics, and safety of Replagal produced by two manufacturing processes in Fabr…
-
CLINICAL_TRIALS trial_result · 2014-03-18Clinical Trial Update: Paricalcitol in Fabry Disease [NA]
Clinical trial update on paricalcitol in Fabry disease; the study status is completed.
-
CLINICAL_TRIALS withdrawal · 2014-02-05Clinical Trial Update: A Study in Patients With Fabry Disease Who Are on Chronic Hemodialysis Therapy for Treatment of End-stage Renal Insufficiency. [Phase 4]
A Phase 4 study evaluating treatment for Fabry disease in patients on chronic hemodialysis has been withdrawn.
-
CLINICAL_TRIALS trial_result · 2013-12-18Clinical Trial Update: A Phase I, Randomized, Single-Blind, Four-Period Cross-Over, Placebo-Controlled, Dose-Escalation Study to Evaluate the Safety and Pharmacokinetics of Single Oral Doses of GR181413A/AT1001 in Healthy Japanese Subjects [Phase 1]
A Phase 1, randomized, single-blind, four-period cross-over, placebo-controlled, dose-escalation study in healthy Japanese subjects evaluati…
-
CLINICAL_TRIALS trial_result · 2013-12-18Clinical Trial Update: Migalastat Food Effect Study [Phase 1]
Phase 1 study assessing the effect of food on migalastat in Fabry disease has completed. The update reports results from the food-effect tri…
-
CLINICAL_TRIALS trial_result · 2013-12-18Clinical Trial Update: A Study to Assess the Absorption, Metabolism and Excretion of Migalastat Hydrochloride (AT1001-014) [Phase 1]
Phase 1 study update assessing how migalastat hydrochloride is absorbed, metabolized, and excreted in Fabry disease. The trial is sponsored …
-
CLINICAL_TRIALS trial_result · 2013-12-04Clinical Trial Update: A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease [Phase 3]
Phase 3 clinical trial update reporting on the safety and efficacy of Fabrazyme for Fabry disease; study completed.
-
CLINICAL_TRIALS trial_result · 2013-11-19Clinical Trial Update: The Fabrazyme® and Arbs and ACE Inhibitor Treatment (FAACET) Study
This completed FAACET study evaluated Fabrazyme in combination with ARB/ACE inhibitor therapy in Fabry disease patients with proteinuria. Th…
-
CLINICAL_TRIALS trial_result · 2013-11-19Clinical Trial Update: Antiproteinuric Agents and Fabry Disease
This clinical trial update reports a completed study evaluating antiproteinuric agents in Fabry disease with proteinuria.