Gaucher Disease
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Sign up freeTop drugs studied for this disease
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FDA approval · 2013-06-03FDA Approved: VPRIV — Supplemental Application (Takeda Pharmaceuticals America, Inc.)
The FDA approved a supplemental application for VPRIV (velaglucerase alfa) from Takeda Pharmaceuticals America, Inc.
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FDA approval · 2013-03-26FDA Approved: VPRIV — Supplemental Application (Takeda Pharmaceuticals America, Inc.)
The FDA approved a supplemental application for VPRIV (velaglucerase alfa) submitted by Takeda Pharmaceuticals America, Inc.
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EMA withdrawal · 2012-11-23EMA Refused: Elelyso (taliglucerase alfa) — Pfizer Ltd.
The EMA refused the marketing authorization for Elelyso (taliglucerase alfa) by Pfizer Ltd., in the Gaucher disease indication.
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CLINICAL_TRIALS research · 2012-10-30Clinical Trial Update: Identification of Undiagnosed Gaucher Disease [NA]
A clinical trial update from Brigham and Women's Hospital on identifying undiagnosed Gaucher disease; the current status is unknown.
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CLINICAL_TRIALS trial_result · 2012-07-26Clinical Trial Update: A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher Disease [Phase 3]
Phase III trial evaluating plant cell expressed GCD for Gaucher disease has completed with results posted in 2012.
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FDA approval · 2012-05-01FDA Approved: ELELYSO — Original Application (Pfizer Laboratories Div Pfizer Inc)
FDA approved ELELYSO (taliglucerase alfa) from Pfizer as the original application for Gaucher disease. This approval introduced the first pl…
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CLINICAL_TRIALS trial_result · 2011-12-02Clinical Trial Update: Macrophagic Activation and Immunophenotypic Profile of Patients With Gaucher's Disease [NA]
A completed clinical trial investigating macrophagic activation and immunophenotypic profiles in patients with Gaucher disease, sponsored by…
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FDA label_change · 2010-11-23FDA Approved: ZAVESCA — Supplemental Application (Actelion Pharmaceuticals US, Inc.)
The FDA approved a supplemental application for ZAVESCA (miglustat) filed by Actelion Pharmaceuticals US, Inc.
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CLINICAL_TRIALS trial_result · 2010-09-02Clinical Trial Update: Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With Imiglucerase [Phase 2, Phase 3]
A phase 2/3 clinical trial evaluating GA-GCB enzyme replacement therapy in Gaucher disease patients previously treated with imiglucerase has…
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CLINICAL_TRIALS trial_result · 2010-08-19Clinical Trial Update: Study to Evaluate Blood Cell Lines From Patients With Gaucher Disease
A completed study sponsored by Amicus Therapeutics investigated blood cell lines from Gaucher disease patients to assess cellular characteri…
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FDA approval · 2010-02-26FDA Approved: VPRIV — Original Application (Takeda Pharmaceuticals America, Inc.)
The FDA approved VPRIV (velaglucerase alfa) from Takeda as the original approval for the treatment of Gaucher disease.
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EMA guidance · 2009-10-22Updated temporary treatment recommendations for Cerezyme
EMA updated temporary treatment recommendations for Cerezyme (imiglucerase) related to its use, dosing, and monitoring guidelines.
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CLINICAL_TRIALS trial_result · 2009-09-02Clinical Trial Update: Safety and Efficacy of Cerezyme® Infusions Every 4 Weeks Versus Every 2 Weeks in Type 1 Gaucher Disease [Phase 4]
A Phase 4 study comparing safety and efficacy of Cerezyme infusions every 4 weeks versus every 2 weeks in Type 1 Gaucher disease.
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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 · 2008-03-04Clinical Trial Update: PEG-Glucocerebrosidase for the Treatment of Gaucher Disease [Phase 1]
Completion of Phase 1 clinical trials evaluating PEG-Glucocerebrosidase for Gaucher disease.
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CLINICAL_TRIALS trial_result · 2008-03-04Clinical Trial Update: Gene Therapy for Gaucher's and Fabry Disease Using Viruses and Blood-Forming Cells [Phase 1]
A Phase 1 clinical trial update on gene therapy for Gaucher disease and Fabry disease using viral vectors and blood-forming cells has comple…
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FDA approval · 2008-02-29FDA Approved: ZAVESCA — Supplemental Application (Actelion Pharmaceuticals US, Inc.)
FDA issued a supplemental approval for ZAVESCA (miglustat) from Actelion Pharmaceuticals US, Inc.
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CLINICAL_TRIALS trial_result · 2006-12-05Clinical Trial Update: Phase I Single Dose-Escalation Safety Study of Human Glucocerebrosidase (prGCD) [Phase 1]
Phase I single-dose escalation safety study of prGCD (human glucocerebrosidase) in Gaucher disease; the trial has completed.