Fabry Disease
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CLINICAL_TRIALS trial_result · 2013-10-10Clinical Trial Update: Replagal Enzyme Replacement Therapy for Children With Fabry Disease [Phase 2]
Phase 2 trial update for Replagal enzyme replacement therapy in children with Fabry disease; study completed with results posted in 2013.
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CLINICAL_TRIALS research · 2013-06-27Clinical Trial Update: Pulmonary Disease and Exercise Tolerance in Boys With Fabry Disease
Clinical trial update examining pulmonary disease and exercise tolerance in boys with Fabry disease; the trial status is unknown.
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CLINICAL_TRIALS trial_result · 2013-04-04Clinical Trial Update: Prevalence of Fabry's Disease in a Population of Patients With Chronic Pains [NA]
This completed clinical trial evaluated the prevalence of Fabry disease in a population of patients with chronic pain.
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CLINICAL_TRIALS research · 2013-03-01Clinical Trial Update: Non-invasive Assessment of Intraocular Pressure in MPS by Use of the Ocular Response Analyzer.
This clinical trial update describes a non-invasive method to assess intraocular pressure in patients with Mucopolysaccharidosis using the O…
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CLINICAL_TRIALS research · 2012-06-14Clinical Trial Update: Fabry and Stroke Epidemiological Protocol (FASEP): Risk Factors In Ischemic Stroke Patients With Fabry Disease
An epidemiological protocol update (FASEP) examining risk factors for ischemic stroke in patients with Fabry disease.
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CLINICAL_TRIALS research · 2012-04-12Clinical Trial Update: Anderson-Fabry Disease in Chronic Kidney Disease Patients Not on Renal Replacement Therapy
This completed observational study assesses the prevalence of Fabry disease in chronic kidney disease patients not on renal replacement ther…
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CLINICAL_TRIALS research · 2012-01-12Clinical Trial Update: Belgian Screening Project for the Detection of Anderson-Fabry Disease in Hypertrophic Cardiomyopathy
The study reports completion of a Belgian screening project to detect Fabry disease in individuals with hypertrophic cardiomyopathy.
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CLINICAL_TRIALS research · 2011-09-28Clinical Trial Update: Myocardial Affectation in Patients With Fabry Disease Without Phenotypic Manifestation. Diagnostic Value of Biomarkers
An update on a trial examining myocardial involvement in Fabry disease patients without phenotypic manifestations and evaluating the diagnos…
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CLINICAL_TRIALS trial_result · 2011-08-17Clinical Trial Update: An Open-Label Maintenance Study of the Enzyme Replacement Therapy Replagal in Patients With Fabry Disease [Phase 1]
An open-label Phase 1 maintenance study of Replagal (agalsidase alfa) for Fabry disease has been completed, according to the trial record.
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CLINICAL_TRIALS research · 2011-07-28Clinical Trial Update: Viennese Prevalence Study of Anderson-Fabry Disease
This entry reports an ongoing clinical trial conducted by the Medical University of Vienna to study the prevalence of Fabry disease, includi…
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CLINICAL_TRIALS trial_result · 2010-12-15Clinical Trial Update: A Study of the Safety and Efficacy of Fabrazyme (Agalsidase Beta) as Compared to Placebo in Patients With Advanced Fabry Disease [Phase 4]
Phase 4 clinical trial comparing Fabrazyme (agalsidase beta) to placebo in patients with advanced Fabry disease completed; results were post…
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CLINICAL_TRIALS trial_result · 2010-08-18Clinical Trial Update: A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease [Phase 4]
Phase 4 trial evaluating the safety and efficacy of Fabrazyme in patients with Fabry disease; results were posted in 2010.
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EMA guidance · 2010-07-06European Medicines Agency updates treatment recommendations because of continued Fabrazyme shortage
EMA updates treatment recommendations due to ongoing Fabrazyme shortage, adjusting guidance on management for patients requiring enzyme repl…
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FDA label_change · 2010-05-14FDA Approved: FABRAZYME — Supplemental Application (Genzyme Corporation)
FDA approved a supplemental application for FABRAZYME (agalsidase beta) from Genzyme Corporation.
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EMA guidance · 2010-04-23European Medicines Agency gives new temporary treatment recommendations for Fabrazyme
EMA provides new temporary treatment recommendations for Fabrazyme, outlining updated dosing or usage guidance while further evaluations con…
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CLINICAL_TRIALS trial_result · 2009-12-21Clinical Trial Update: Fabry : National Initiative of Screening [NA]
The document reports completion of a Fabry disease screening trial within ischemic stroke patients under the National Initiative of Screenin…
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EMA guidance · 2009-09-25Supply shortage of Fabrazyme – updated treatment recommendations required for adult male patients
EMA updates treatment recommendations for Fabrazyme due to supply shortage, focusing on adult male patients.
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EMA guidance · 2009-06-25Supply shortages of Cerezyme and Fabrazyme - priority access for patients most in need of treatment recommended
EMA recommends priority access to Cerezyme and Fabrazyme during supply shortages to ensure treatment for patients most in need.
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CLINICAL_TRIALS trial_result · 2009-06-16Clinical Trial Update: A Study of Fabrazyme in Pediatric Patients With Fabry Disease [Phase 2]
This clinical trial update summarizes Phase 2 results from a pediatric study of Fabrazyme in patients with Fabry disease.
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CLINICAL_TRIALS trial_result · 2009-04-02Clinical Trial Update: A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease [Phase 2]
Phase 2 study evaluating Fabrazyme for Fabry disease assessed safety and efficacy; results were posted in 2009.
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CLINICAL_TRIALS trial_result · 2009-02-05Clinical Trial Update: Severe Renal Disease Study in Fabry Patients Treated With Fabrazyme [Phase 2]
Phase 2 trial of Fabrazyme in Fabry disease patients with severe renal disease was terminated; results were posted in 2009.
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FDA approval · 2008-12-17FDA Approved: FABRAZYME — Supplemental Application (Genzyme Corporation)
The FDA approved a supplemental application for Fabrazyme (agalsidase beta) submitted by Genzyme Corporation. This approval authorizes chang…
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CLINICAL_TRIALS trial_result · 2008-03-04Clinical Trial Update: Alternative Dosing and Regimen of Replagal to Treat Fabry Disease [Phase 2]
Phase 2 trial evaluating alternative dosing regimens of Replagal (agalsidase alfa) for Fabry disease has been completed.
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CLINICAL_TRIALS trial_result · 2008-03-04Clinical Trial Update: Alpha-Galactosidase A Replacement Therapy for Fabry Disease [Phase 2]
A Phase 2 trial of Alpha-Galactosidase A replacement therapy for Fabry disease has completed.
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CLINICAL_TRIALS trial_result · 2008-03-04Clinical Trial Update: An Open-Label Clinical Trial of Replagal Enzyme Therapy in Children Ages 7-17 Years With Fabry Disease [Phase 2]
An open-label Phase 2 trial evaluating agalsidase alfa (Replagal) enzyme therapy in children ages 7-17 with Fabry disease; the study status …