Gaucher Disease
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS trial_result · 2016-09-28Clinical Trial Update: An Open-Label, Dose Escalation Study to Evaluate the Safety and the Pharmacokinetics of Oral PRX-112 [Phase 2]
This is an update on a Phase 2 open-label dose-escalation study evaluating the safety and pharmacokinetics of the oral drug PRX-112 in Gauch…
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CLINICAL_TRIALS trial_result · 2016-09-22Clinical Trial Update: Bone Response to Enzyme Replacement in Gaucher's Disease [Phase 2]
A Phase 2 trial evaluating bone response to enzyme replacement therapy in Gaucher's disease has completed.
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CLINICAL_TRIALS trial_result · 2016-01-18Clinical Trial Update: A Multicenter Extension Study of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease [Phase 3]
Multicenter extension study of taliglucerase alfa in pediatric Gaucher disease patients completed with posted results.
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CLINICAL_TRIALS trial_result · 2016-01-08Clinical Trial Update: Auto-antibodies Prevalence and CD1 Role in Gaucher Disease
This clinical trial update reports results from a completed study evaluating the prevalence of auto-antibodies and the role of CD1 in Gauche…
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CLINICAL_TRIALS trial_result · 2015-12-14Clinical Trial Update: A Multicenter Extension Study of Taliglucerase Alfa in Adult Subjects With Gaucher Disease [Phase 3]
A multicenter extension study of taliglucerase alfa in adults with Gaucher disease has completed phase 3.
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CLINICAL_TRIALS trial_result · 2015-12-14Clinical Trial Update: Multicenter Extension Study of Velaglucerase Alfa in Japanese Patients With Gaucher Disease [Phase 3]
Phase 3 multicenter extension study of velaglucerase alfa in Japanese patients with Gaucher disease has completed with results posted.
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CLINICAL_TRIALS trial_result · 2015-05-27Clinical Trial Update: Taste Evaluation of Different Liquid Formulations With Eliglustat [Phase 1]
A Phase 1 study evaluating taste and palatability of different liquid formulations of eliglustat in Gaucher disease.
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CLINICAL_TRIALS trial_result · 2015-03-25Clinical Trial Update: Phase II Randomized Study of Alendronate Sodium for Osteopenia in Patients With Gaucher's Disease [Phase 2]
Phase II randomized trial of alendronate for osteopenia in Gaucher disease patients completed.
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CLINICAL_TRIALS trial_result · 2015-02-04Clinical Trial Update: MR Spectroscopy and Disease Severity Score for Gaucher in Pediatric Population
This pediatric Gaucher disease trial evaluating MR spectroscopy and a disease severity score has completed, sponsored by the Children's Hosp…
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CLINICAL_TRIALS trial_result · 2014-11-11Clinical Trial Update: A Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease [Phase 4]
Phase 4 trial evaluating two dose levels of taliglucerase alfa in pediatric Gaucher disease subjects; completed with results posted in 2014.
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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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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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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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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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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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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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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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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.
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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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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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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…