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 research · 2026-04-21Clinical Trial Update: A Gaucher Disease Gene Therapy Trial With FLT201 [Phase 3]
A Phase 3 Gaucher disease type 1 gene therapy trial (FLT201) is currently recruiting.
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CLINICAL_TRIALS research · 2026-04-15Clinical Trial Update: A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED) [Phase 1, Phase 2]
Clinical trial PROCEED is a Phase 1/2 study evaluating PR001 (LY3884961) for peripheral manifestations of Gaucher disease, and the trial is …
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CLINICAL_TRIALS research · 2026-04-01Clinical Trial Update: Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE) [Phase 1, Phase 2]
Phase 1/2 clinical trial update for PR001 in infants with Gaucher disease type 2 (PROVIDE), currently active and not recruiting.
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CLINICAL_TRIALS research · 2026-03-19Clinical Trial Update: Oxidative Stress and Inflammatory Biomarkers in Gaucher Disease
A recruiting clinical trial is investigating oxidative stress and inflammatory biomarkers in Gaucher disease Type I. The sponsor is the Univ…
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CLINICAL_TRIALS research · 2026-03-17Clinical Trial Update: PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders) [Phase 1]
Phase 1 prenatal enzyme replacement therapy trial PEARL is currently recruiting for multiple lysosomal storage disorders including MPS I, MP…
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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 research · 2026-02-04Clinical Trial Update: A Clinical Study for the Treatment of Pediatric and Adolescent Patients With Type 1 Gaucher Disease [EARLY_Phase 1]
This is an EARLY Phase 1 clinical study for the treatment of pediatric and adolescent patients with Type 1 Gaucher disease; the trial is cur…
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CLINICAL_TRIALS research · 2026-01-26Clinical Trial Update: A Clinical Study Evaluating LY-M001 Injection in the Treatment of Adult Patients With Type I Gaucher Disease [Phase 1, Phase 2]
Phase 1/2 clinical study evaluating LY-M001 injection in adults with Gaucher disease type 1 is currently recruiting. The sponsor is Lingyi B…
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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 research · 2026-01-08Clinical Trial Update: MAGNETIC RESONANCE SPECTROSCOPY BIOMARKERS IN TYPE 3 GAUCHER DISEASE (GD3)
An ongoing University of Minnesota trial is investigating magnetic resonance spectroscopy biomarkers in Gaucher disease type 3 and is curren…
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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 research · 2025-11-04Clinical Trial Update: Long Term Follow-up Study of Type-1 Gaucher Subjects Post FLT201 Dose (GALILEO-2) [Phase 1, Phase 2]
Long-term follow-up GALILEO-2 study of FLT201 in Gaucher disease type 1 is a Phase 1/2 trial sponsored by Spur Therapeutics, with status lis…
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CLINICAL_TRIALS research · 2025-10-20Clinical Trial Update: Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3 [Phase 3]
Status update on a Phase 3 trial evaluating the efficacy and safety of venglustat for Gaucher disease type 3; the study is currently active …
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CLINICAL_TRIALS research · 2025-03-26Clinical Trial Update: Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension [Phase 2]
Clinical trial update for a Phase 2 study of venglustat with Cerezyme in adults with Gaucher disease type 3, including a venglustat monother…
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CLINICAL_TRIALS research · 2025-02-14Clinical Trial Update: A Study of the Safety and Preliminary Efficacy of LY-M001 Injection in the Treatment of Adult Patients With Gaucher Disease Type I [NA]
This document reports an ongoing clinical trial evaluating the safety and preliminary efficacy of LY-M001 injection in adults with Gaucher d…
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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 research · 2024-08-06Clinical Trial Update: Gaucherite - A Study to Stratify Gaucher Disease
This entry describes the Gaucherite study to stratify Gaucher disease (types I and III), which is currently recruiting under Cambridge Unive…
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FDA approval · 2024-07-10FDA Approved: CEREZYME — Supplemental Application (Genzyme Corporation)
FDA approved a supplemental application for Cerezyme (imiglucerase) from Genzyme Corporation.
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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 research · 2022-07-27Clinical Trial Update: Phase 1/2 Study of CAN103 in Subjects With Gaucher Disease [Phase 1, Phase 2]
Phase 1/2 clinical trial update for CAN103 in Gaucher disease (Type 1 and Type 3) by CANbridge.
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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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FDA approval · 2018-08-29FDA Approved: CERDELGA — Supplemental Application (Genzyme Corporation)
The FDA approved a supplemental application for Cerdelga (eliglustat) filed by Genzyme Corporation.