Gaucher Disease
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS trial_result · 2014-09-03Clinical Trial Update: A Study of the Efficacy and Safety of Eliglustat Tartrate (Genz-112638) in Type 1 Gaucher Patients [Phase 2]
A Phase 2 trial evaluating the efficacy and safety of eliglustat tartrate in adults with type 1 Gaucher disease has completed, with results …
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FDA approval · 2014-08-27FDA Approved: ELELYSO — Supplemental Application (Pfizer Laboratories Div Pfizer Inc)
The FDA approved a supplemental application for ELELYSO (taliglucerase alfa) from Pfizer Laboratories Div Pfizer Inc.
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CLINICAL_TRIALS trial_result · 2014-08-04Clinical Trial Update: Study of Velaglucerase Alfa Enzyme Replacement Therapy in Japanese Patients With Gaucher Disease [Phase 3]
Phase 3 trial of velaglucerase alfa enzyme replacement therapy in Japanese patients with Gaucher disease completed; results posted in 2014.
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CLINICAL_TRIALS trial_result · 2014-07-15Clinical Trial Update: Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial [Phase 3]
Phase 3 trial update for plant cell expressed recombinant human glucocerebrosidase in Gaucher disease has completed with results posted.
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FDA approval · 2014-07-11FDA Approved: VPRIV — Supplemental Application (Takeda Pharmaceuticals America, Inc.)
FDA has approved a supplemental application for VPRIV (velaglucerase alfa) from Takeda Pharmaceuticals America, Inc.
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FDA approval · 2014-06-24FDA Approved: ELELYSO — Supplemental Application (Pfizer Laboratories Div Pfizer Inc)
The FDA approved a supplemental application for ELELYSO (taliglucerase alfa) submitted by Pfizer Laboratories Div Pfizer Inc.
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CLINICAL_TRIALS trial_result · 2014-06-23Clinical Trial Update: Open-Label Extension Study Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A (ERT) [Phase 1, Phase 2]
Open-label extension study evaluating long-term safety of DRX008A (ERT) in patients with Type 1 Gaucher disease completed with results poste…
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FDA approval · 2014-06-18FDA 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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CLINICAL_TRIALS trial_result · 2014-06-05Clinical Trial Update: Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase [Phase 3]
Phase 3 clinical trial update on switching from imiglucerase to plant cell expressed recombinant human glucocerebrosidase in Gaucher disease…
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CLINICAL_TRIALS trial_result · 2014-05-23Clinical Trial Update: Cellular and Molecular Mechanisms Governing Bone Marrow Stem Cells in Gaucher Disease [NA]
A completed clinical trial investigating the cellular and molecular mechanisms governing bone marrow stem cells in Gaucher disease.
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FDA label_change · 2014-05-14FDA Approved: ELELYSO — Supplemental Application (Pfizer Laboratories Div Pfizer Inc)
The FDA approved a supplemental application for ELELYSO (taliglucerase alfa) filed by Pfizer Laboratories Div Pfizer Inc. The approval resul…
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EMA guidance · 2014-05-14European Medicines Agency and US Food and Drug Administration release joint proposal to facilitate clinical investigation of new medicines for Gaucher disease in children
The EMA and FDA released a joint proposal to facilitate clinical investigations of new medicines for Gaucher disease in children.
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CLINICAL_TRIALS trial_result · 2014-04-07Clinical Trial Update: Safety and Pharmacokinetics of Oral PRX-112 in Gaucher Disease Patients [Phase 1]
A Phase 1 study assessing the safety and pharmacokinetics of oral PRX-112 in Gaucher disease patients has been completed.
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FDA approval · 2014-02-27FDA Approved: CEREZYME — Supplemental Application (Genzyme Corporation)
FDA approved a supplemental application for Cerezyme (imiglucerase) from Genzyme Corporation. This action concerns the approval of an additi…
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FDA approval · 2014-02-11FDA Approved: VPRIV — Supplemental Application (Takeda Pharmaceuticals America, Inc.)
FDA approved a supplemental application for VPRIV (velaglucerase alfa) submitted by Takeda Pharmaceuticals America, Inc. The document indica…
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CLINICAL_TRIALS trial_result · 2014-02-04Clinical Trial Update: A Switch-Over Study of the Safety and Efficacy of ISU302 in Patients With Type 1 Gaucher Disease
A completed switch-over clinical trial evaluating the safety and efficacy of ISU302 in patients with Type 1 Gaucher disease.
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FDA approval · 2014-01-13FDA Approved: ELELYSO — Supplemental Application (Pfizer Laboratories Div Pfizer Inc)
The FDA has approved a supplemental application for Elelyso (taliglucerase alfa) from Pfizer Laboratories Div Pfizer Inc.
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FDA approval · 2013-11-21FDA Approved: VPRIV — Supplemental Application (Takeda Pharmaceuticals America, Inc.)
The FDA approved a supplemental application for VPRIV (velaglucerase alfa) submitted by Takeda Pharmaceuticals America, Inc. This action rel…
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FDA approval · 2013-10-30FDA Approved: CEREZYME — Supplemental Application (Genzyme Corporation)
The FDA approved a supplemental application for Cerezyme (imiglucerase) from Genzyme Corporation.
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FDA approval · 2013-10-02FDA Approved: VPRIV — Supplemental Application (Takeda Pharmaceuticals America, Inc.)
The FDA approved a supplemental application for VPRIV (velaglucerase alfa) submitted by Takeda Pharmaceuticals America, Inc., approving chan…
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CLINICAL_TRIALS trial_result · 2013-09-27Clinical Trial Update: Intra-monocyte Imiglucerase Kinetics in Gaucher Disease [Phase 2]
Phase 2 trial update examining intra-monocyte kinetics of imiglucerase in Gaucher disease.
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CLINICAL_TRIALS trial_result · 2013-08-02Clinical Trial Update: Intravenous N-acetylcysteine for the Treatment of Gaucher's Disease and Parkinson's Disease [Phase 1]
Phase 1 trial investigating intravenous N-acetylcysteine for Parkinson's disease and Gaucher's disease has completed with results posted in …
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FDA approval · 2013-07-31FDA Approved: CEREZYME — Supplemental Application (Genzyme Corporation)
The FDA approved a supplemental application for Cerezyme (imiglucerase) submitted by Genzyme Corporation. This action finalizes changes to t…
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CLINICAL_TRIALS research · 2013-07-12Clinical Trial Update: Assessment Of Liver and Spleen Fibrosis in Patients With Gaucher Disease Using Fibroscan [NA]
Clinical trial update describing an observational assessment of liver and spleen fibrosis in patients with Gaucher disease using FibroScan.
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CLINICAL_TRIALS trial_result · 2013-06-19Clinical Trial Update: A Study of the Tolerability, Safety, and Pharmacokinetics of ISU302 in Healthy Volunteers [Phase 1]
Phase 1 trial update reporting tolerability, safety, and pharmacokinetics of ISU302 in healthy volunteers; the study has completed.