Gaucher Disease
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS trial_result · 2025-07-22Clinical Trial Update: Long Term Follow-Up for Safety of AVR-RD-02
This is a long-term follow-up safety study of AVR-RD-02 for Gaucher disease, currently listed as active but not recruiting.
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CLINICAL_TRIALS research · 2025-06-26Clinical Trial Update: Omics Gaucher Study: Multiomic Approach
A multiomic study on Gaucher disease led by CENTOGENE is currently active but not recruiting.
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EMA approval · 2025-06-25EMA Authorised: Miglustat Dipharma (miglustat) — Dipharma Arzneimittel GmbH
The EMA authorised Miglustat Dipharma (miglustat) by Dipharma Arzneimittel GmbH for Gaucher disease, granting a European marketing authoriza…
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EMA approval · 2025-06-24EMA Authorised: Miglustat Gen.Orph (miglustat) — Gen.Orph
The EMA authorised Miglustat Gen.Orph for the treatment of Gaucher disease, marking a new regulatory approval for this product in the EU.
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CLINICAL_TRIALS trial_result · 2025-05-31Clinical Trial Update: The GBA Multimodal Study in Parkinson's Disease
The GBA Multimodal Study in Parkinson's Disease is an ongoing clinical trial investigating GBA gene mutations in Parkinson's disease and is …
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EMA approval · 2025-05-16EMA Authorised: Cerdelga (Eliglustat (tartrate)) — Sanofi B.V.
The European Medicines Agency authorised Cerdelga (eliglustat tartrate) for Gaucher disease, with orphan medicine designation; decision date…
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CLINICAL_TRIALS research · 2025-03-03Clinical Trial Update: Drug Discovery for Parkinson's With Mutations in the GBA Gene
This clinical trial update describes a study focused on drug discovery for Parkinson's disease in patients with Gaucher disease or GBA gene …
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CLINICAL_TRIALS trial_result · 2025-02-26Clinical Trial Update: A Study of Velaglucerase Alfa (VPRIV) in Chinese Children, Teenagers, and Adults With Type 1 Gaucher Disease [Phase 3]
Phase 3 trial update of Velaglucerase alfa (VPRIV) in Chinese children, adolescents, and adults with Type 1 Gaucher disease has completed, w…
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CLINICAL_TRIALS trial_result · 2025-01-10Clinical Trial Update: Survey Study for Velaglucerase Alfa (VPRIV) in Japan
A survey study evaluating velaglucerase alfa (VPRIV) in Gaucher disease patients in Japan has completed, and the results have been posted.
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FDA approval · 2024-12-19FDA Approved: CEREZYME — Supplemental Application (Genzyme Corporation)
The FDA approved a supplemental application for Cerezyme (imiglucerase) submitted by Genzyme Corporation.
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CLINICAL_TRIALS trial_result · 2024-12-17Clinical Trial Update: A Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher Disease [Phase 4]
A Phase 4 multicenter trial evaluating the safety and efficacy of taliglucerase alfa in subjects with Gaucher disease, Type 3, has completed…
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CLINICAL_TRIALS trial_result · 2024-12-11Clinical Trial Update: A Study Measuring Substances Potentially Indicating Bone Problems in Adults With Type 1 Gaucher Condition
Takeda-sponsored study assessing substances potentially indicating bone problems in adults with Gaucher disease has completed.
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CLINICAL_TRIALS research · 2024-11-19Clinical Trial Update: Implementation and Evaluation of a Rare Disease Algorithm to Identify Persons at Risk of Gaucher Disease Using Data From Electronic Health Records (EHRs) in the United States (Project Searchlight) [NA]
A completed clinical trial project evaluating a rare disease algorithm to identify Gaucher disease risk using US electronic health records.
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CLINICAL_TRIALS research · 2024-07-26Clinical Trial Update: Efficacy and Safety of Eliglustat in Chinese Pediatric Patients With Gaucher Disease Type 1 and Type 3 [Phase 2]
Phase 2 clinical trial evaluating the efficacy and safety of eliglustat in Chinese pediatric patients with Gaucher disease types 1 and 3; th…
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FDA approval · 2024-07-10FDA 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.
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FDA approval · 2024-07-10FDA Approved: VPRIV — Supplemental Application (Takeda Pharmaceuticals America, Inc.)
FDA approved a supplemental application for VPRIV (velaglucerase alfa) by Takeda Pharmaceuticals America, Inc.
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CLINICAL_TRIALS trial_result · 2024-01-18Clinical Trial Update: Role of Vitamin E in Gaucher Disease Patients [Phase 3]
Phase 3 clinical trial evaluating role of Vitamin E in Gaucher disease patients has completed.
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CLINICAL_TRIALS trial_result · 2024-01-18Clinical Trial Update: Lentiviral Vector Gene Therapy - The Guard1 Trial of AVR-RD-02 for Subjects With Type 1 Gaucher Disease [Phase 1, Phase 2]
A phase 1/2 trial of AVR-RD-02 using a lentiviral vector in Gaucher disease has been terminated with results posted.
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CLINICAL_TRIALS trial_result · 2023-12-21Clinical Trial Update: A Study of Enzyme Replacement Therapy (VPRIV) in People With Type 1 Gaucher Disease Who Were Previously Treated With Substrate Reduction Therapy [Phase 4]
Phase 4 trial updating enzyme replacement therapy VPRIV in people with Gaucher disease previously treated with substrate reduction therapy; …
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CLINICAL_TRIALS withdrawal · 2023-08-09Clinical Trial Update: An Efficacy and Safety Study of AVR-RD-02 Compared to Enzyme Replacement Therapy for Treatment of Gaucher Disease Type 3 [Phase 2, Phase 3]
A clinical trial evaluating AVR-RD-02 for Gaucher disease type 3 was withdrawn after being studied in Phase 2/3.
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FDA approval · 2023-05-09FDA 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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FDA approval · 2022-08-30FDA Approved: ELELYSO — Supplemental Application (Pfizer Laboratories Div Pfizer Inc)
FDA approved a supplemental application for ELELYSO (taliglucerase alfa) from Pfizer Laboratories Div Pfizer Inc.
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CLINICAL_TRIALS trial_result · 2022-07-15Clinical Trial Update: Phase 3b Study to Evaluate Skeletal Response to Eliglustat in Adult Patients Who Completed Phase 2 or Phase 3 Studies [Phase 3]
Phase 3b trial update evaluating skeletal response to eliglustat in adults who completed prior studies for Gaucher disease; status completed…
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CLINICAL_TRIALS trial_result · 2022-02-01Clinical Trial Update: Study of the Effect of Velaglucerase Alfa (VPRIV®) on Bone-related Pathology in Treatment-naïve Participants With Type 1 Gaucher Disease [Phase 4]
A Phase 4 study evaluating velaglucerase alfa (VPRIV) on bone-related pathology in treatment-naïve adults with Type 1 Gaucher disease has co…
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FDA approval · 2021-12-22FDA Approved: CEREZYME — Supplemental Application (Genzyme Corporation)
The FDA approved a supplemental application for Cerezyme (imiglucerase) submitted by Genzyme Corporation. The action updates the product's a…