Gaucher Disease Type
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS trial_result · 2026-06-02Clinical Trial Results: A Gene Therapy Study in Patients With Gaucher Disease Type 1 [Phase 1]
Phase 1 gene therapy trial in Gaucher disease type 1 completed; results posted.
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CLINICAL_TRIALS trial_result · 2026-02-18Clinical Trial Update: Role of Oxidative Stress and Inflammation in Type 1 Gaucher Disease (GD1) [Phase 2]
Phase 2 clinical trial investigating the role of oxidative stress and inflammation in Gaucher disease type 1 has completed.
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CLINICAL_TRIALS trial_result · 2026-01-14Clinical Trial Update: Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3 [Phase 3]
Phase 3 trial assessing safety and efficacy of eliglustat with or without imiglucerase in pediatric Gaucher disease types 1 and 3; the study…
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CLINICAL_TRIALS trial_result · 2025-11-04Clinical Trial Update: A Gene Therapy Study in Patients With Gaucher Disease Type 1 [Phase 1]
A Phase 1 gene therapy study for Gaucher disease type 1 has completed, with Spur Therapeutics listed as the sponsor and the trial status as …
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CLINICAL_TRIALS trial_result · 2024-10-04Clinical Trial Update: Effect of Enzyme Replacement Therapy on Cardiac Function in Children With Gaucher Disease Type 3
An ongoing clinical trial is evaluating the impact of enzyme replacement therapy on cardiac function in children with Gaucher disease type 3…
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CLINICAL_TRIALS trial_result · 2023-09-22Clinical Trial Update: A Second-generation AI Based Therapeutic Regimen in Patients With Gaucher Disease Treated With Enzyme Replacement Therapy. [Phase 2]
A Phase 2 clinical trial reports on a second-generation AI-based therapeutic regimen for Gaucher disease type 1 in patients already treated …
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CLINICAL_TRIALS trial_result · 2023-02-14Clinical Trial Update: Ambroxol Therapy for Patients With Type 1 Gaucher Disease and Suboptimal Response to Enzyme Replacement Therapy [Phase 2]
Phase 2 trial assessing ambroxol therapy for Gaucher disease type 1 patients with suboptimal response to enzyme replacement therapy has comp…
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CLINICAL_TRIALS trial_result · 2022-10-28Clinical Trial Update: Long Term Impact of Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV) [Phase 4]
Phase 4 clinical trial assessing the long-term impact of rapid intravenous infusion of velaglucerase alfa (VPRIV) for Gaucher disease type 1…
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CLINICAL_TRIALS trial_result · 2021-03-19Clinical Trial Update: Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV) in Treatment-naive Patients With Type 1 Gaucher Disease [Phase 4]
Phase 4 trial update on rapid intravenous infusion of velaglucerase alfa (VPRIV) in treatment-naive adults with Gaucher disease type 1.
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CLINICAL_TRIALS trial_result · 2018-08-15Clinical Trial Update: A Study of AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy [Phase 2]
Phase 2 trial evaluating AT2101 (afegostat tartrate) in adults with Type 1 Gaucher disease already on enzyme replacement therapy has complet…
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CLINICAL_TRIALS trial_result · 2018-08-15Clinical Trial Update: A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher Patients [Phase 2]
Long-term extension study update of AT2101 (Afegostat Tartrate) in patients with Type 1 Gaucher disease completed, with results posted in 20…
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CLINICAL_TRIALS trial_result · 2018-08-15Clinical Trial Update: A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher Disease [Phase 2]
Phase 2 trial update for oral AT2101 (afegostat tartrate) in treatment-naive Gaucher disease patients completed with posted results.
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CLINICAL_TRIALS trial_result · 2017-07-12Clinical Trial Update: Phase III Study of ISU302 in Patients With Type 1 Gaucher Disease [Phase 3]
A Phase 3 trial evaluating ISU302 in Type 1 Gaucher disease has completed enrollment and posted results.
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CLINICAL_TRIALS trial_result · 2015-06-01Clinical Trial Update: Validating a New Severity Score System for Adults With Type 1 Gaucher Disease (GD1)
A completed clinical trial aimed at validating a new severity score system for adults with Gaucher disease type 1.
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CLINICAL_TRIALS trial_result · 2015-03-05Clinical Trial Update: A Multicenter Study of the Efficacy of Cerezyme in Testing Skeletal Disease in Patients With Type I Gaucher Disease. [Phase 4]
A Phase 4 multicenter trial evaluating the efficacy of Cerezyme for skeletal disease in Gaucher disease type I has been completed.
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CLINICAL_TRIALS trial_result · 2014-11-19Clinical Trial Update: Comparison of BMD Measurement by DEXA to BeamMed Speed-of-Sound Measurement at Forearm in Patients With Gaucher Disease [NA]
This clinical trial update reports a completed study comparing bone mineral density measurements obtained by DEXA versus BeamMed speed-of-so…
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CLINICAL_TRIALS trial_result · 2014-09-04Clinical Trial Update: A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE) [Phase 3]
This Phase 3 ENCORE trial evaluated switching Gaucher disease type 1 patients who had achieved therapeutic goals on enzyme replacement thera…
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CLINICAL_TRIALS trial_result · 2014-09-03Clinical Trial Update: A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease (ENGAGE) [Phase 3]
Phase 3 ENGAGE trial results for Eliglustat Tartrate in Gaucher disease type 1 were completed, with results posted in 2014.
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CLINICAL_TRIALS trial_result · 2014-01-28Clinical Trial Update: An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease [Phase 3]
Open-label extension study of GA-GCB enzyme replacement therapy in adults with Type 1 Gaucher disease; phase 3 completed with results posted…
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CLINICAL_TRIALS trial_result · 2013-02-15Clinical Trial Update: Clinical Trial of Ambroxol in Patients With Type I Gaucher Disease [Phase 1, Phase 2]
A Phase 1/2 clinical trial of Ambroxol in Gaucher disease type 1 has been suspended.
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CLINICAL_TRIALS trial_result · 2012-05-23Clinical Trial Update: Clinical Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Miglustat in Patients With Stable Type 1 Gaucher Disease [Phase 3]
Miglustat was evaluated in a Phase 3 long-term study for efficacy, safety, and tolerability in patients with stable Gaucher disease type 1. …
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CLINICAL_TRIALS trial_result · 2011-01-04Clinical Trial Update: Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) ERT Compared With Imiglucerase in Type I Gaucher Disease [Phase 3]
Phase 3 trial comparing GA-GCB enzyme replacement therapy to imiglucerase in Type 1 Gaucher disease has completed with results posted.
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CLINICAL_TRIALS trial_result · 2010-09-10Clinical Trial Update: A Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Gaucher Disease [Phase 3]
Phase 3 trial of GA-GCB enzyme replacement therapy for Gaucher disease type 1 has completed with posted results.