Gaucher Disease
📧 Sign up to get email alerts when something new happens for Gaucher Disease.
Sign up freeTop drugs studied for this disease
-
FDA approval · 2020-12-10FDA Approved: VPRIV — Supplemental Application (Takeda Pharmaceuticals America, Inc.)
The FDA approved a supplemental application for VPRIV (velaglucerase alfa) submitted by Takeda Pharmaceuticals America, Inc.
-
FDA approval · 2020-11-03FDA 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.
-
FDA approval · 2019-11-06FDA Approved: VPRIV — Supplemental Application (Takeda Pharmaceuticals America, Inc.)
The FDA approved a supplemental application for VPRIV (velaglucerase alfa) submitted by Takeda Pharmaceuticals America, Inc., authorizing re…
-
FDA approval · 2019-10-21FDA Approved: ELELYSO — Supplemental Application (Pfizer Laboratories Div Pfizer Inc)
FDA approved a supplemental application for ELELYSO (taliglucerase alfa) submitted by Pfizer Laboratories Div Pfizer Inc, under BLA022458.
-
FDA approval · 2019-07-30FDA Approved: VPRIV — Supplemental Application (Takeda Pharmaceuticals America, Inc.)
FDA approved a supplemental application for VPRIV (velaglucerase alfa) submitted by Takeda Pharmaceuticals America, Inc. This action updates…
- Want full summaries, importance scores, and drug timelines? Upgrade to Plus or Pro.
-
CLINICAL_TRIALS research · 2019-01-23Clinical Trial Update: Biomarker Analysis for GBA Associated Parkinson's Disease
This clinical trial update describes biomarker analysis for Gaucher disease–associated Parkinson's disease (GBA-related). The current status…
-
CLINICAL_TRIALS trial_result · 2018-10-30Clinical Trial Update: Chronic Hepatitis C Treatment in Egyptian Children With Gaucher Disease. [Phase 4]
A Phase 4 clinical trial evaluating chronic hepatitis C treatment in Egyptian children with Gaucher disease is ongoing, with status currentl…
-
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…
-
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…
-
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.
-
CLINICAL_TRIALS research · 2018-07-18Clinical Trial Update: Clinical Audit of Managment of Gausher Disease in Children
This document reports a clinical audit of how Gaucher disease is managed in children, with limited information and an unknown study status.
-
EMA guidance · 2017-07-03EMA and FDA encourage use of innovative approaches in the development of medicines for Gaucher disease
EMA and FDA encourage the use of innovative development approaches to speed up and improve medicines for Gaucher disease.
-
CLINICAL_TRIALS trial_result · 2017-07-02Clinical Trial Update: OGT 918-006: A Phase I/II Randomized, Controlled Study of OGT 918 in Patients With Neuronopathic Gaucher Disease [Phase 2]
This update notes that the Phase II randomized, controlled trial of OGT 918 for Gaucher disease (neuronopathic form) has completed.
-
CLINICAL_TRIALS trial_result · 2017-07-02Clinical Trial Update: Effects of Enzyme Replacement in Gaucher's Disease
Completed clinical trial evaluating enzyme replacement therapy effects in Gaucher disease. The provided snippet does not detail specific res…
-
CLINICAL_TRIALS research · 2017-07-02Clinical Trial Update: A Study to Evaluate and Characterize the Effect of Pharmacological Chemicals on Blood From Patients With Gaucher Disease
This clinical trial update describes a completed study that aimed to evaluate and characterize the effects of pharmacological chemicals on b…
-
CLINICAL_TRIALS trial_result · 2017-04-04Clinical Trial Update: Exploration of Immunity in Gaucher Disease
This is a completed clinical trial exploring immunity in Gaucher disease sponsored by O & O Alpan LLC (NCT01358188).
-
CLINICAL_TRIALS research · 2017-04-04Clinical Trial Update: Investigating Lysosomal Storage Diseases in Minority Groups
A clinical trial update from O & O Alpan LLC examining lysosomal storage disorders in minority populations, including Gaucher disease, Fabry…
-
CLINICAL_TRIALS trial_result · 2017-03-08Clinical Trial Update: A Study of the Effects of Renal Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate [Phase 1]
This Phase 1 study evaluated the effects of renal impairment on the pharmacokinetics and tolerability of eliglustat tartrate in Gaucher dise…
-
CLINICAL_TRIALS trial_result · 2017-02-10Clinical Trial Update: A Study of the Effects of Hepatic Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate [Phase 1]
Phase 1 study evaluating the effect of hepatic impairment on the pharmacokinetics and tolerability of eliglustat tartrate in Gaucher disease…
-
CLINICAL_TRIALS trial_result · 2016-12-02Clinical Trial Update: A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease to Evaluate Once Daily Versus Twice Daily Dosing (EDGE) [Phase 3]
Phase 3 trial comparing once-daily versus twice-daily dosing of eliglustat tartrate in Gaucher disease has completed with results posted.
-
CLINICAL_TRIALS research · 2016-10-28Clinical Trial Update: Thrombocytopathy in Gaucher Disease Patients
An update on a clinical trial investigating thrombocytopathy in Gaucher disease patients; the current status of the study is unknown. No res…
-
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…
-
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.
-
FDA approval · 2016-07-22FDA Approved: CEREZYME — Supplemental Application (Genzyme Corporation)
FDA approved a supplemental Biologics License Application for Cerezyme (imiglucerase) by Genzyme Corporation.
-
FDA approval · 2016-07-07FDA Approved: CEREZYME — Supplemental Application (Genzyme Corporation)
FDA approved a supplemental application for Cerezyme (imiglucerase) submitted by Genzyme Corporation.