Gaucher Disease
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS research · 2026-04-24Clinical Trial Update: Genetic Studies of Lysosomal Storage Disorders
A genetic study of lysosomal storage disorders, including Gaucher disease and potentially Parkinson disease, is currently enrolling by invit…
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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 · 2026-04-14Clinical Trial Update: International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry
An international Gaucher disease registry and pregnancy sub-registry sponsored by Genzyme (Sanofi) is currently recruiting.
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CLINICAL_TRIALS research · 2026-04-06Clinical Trial Update: Extracellular Vesicles as Potential Biomarkers and Therapeutic Target in Gaucher Disease
A clinical trial exploring extracellular vesicles as potential biomarkers and a therapeutic target for Gaucher disease is currently recruiti…
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CLINICAL_TRIALS research · 2026-04-03Clinical Trial Update: A Survey to Assess Participants', Caregivers', and Nurses' Use and Understanding of Educational Material on Velaglucerase Alfa (VPRIV) Home Infusion
A survey is being conducted to evaluate how participants, caregivers, and nurses use and understand educational materials for Velaglucerase …
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CLINICAL_TRIALS research · 2026-02-11Clinical Trial Update: GammaGA: Prevalence of Acid Sphingomyelinase Deficiency Disease (ASMD) and Gaucher Disease in Patients With Monoclonal Gammopathies and/or Multiple Myeloma
Clinical trial update for GammaGA examining how common acid sphingomyelinase deficiency (ASMD) and Gaucher disease are in patients with mono…
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CLINICAL_TRIALS research · 2026-01-14Clinical Trial Update: Institutional Registry of Rare Diseases
An institutional registry for rare diseases sponsored by Hospital Italiano de Buenos Aires is currently recruiting. The registry includes am…
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CLINICAL_TRIALS research · 2025-10-09Clinical Trial Update: Gaucher Disease Outcome Survey (GOS)
The Gaucher Disease Outcome Survey (GOS) is a recruiting clinical trial sponsored by Shire to collect data on Gaucher disease.
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CLINICAL_TRIALS research · 2025-09-23Clinical Trial Update: A Non-Interventional National Study in Pediatric Patients With Unexplained Enlarged Spleen
A non-interventional national pediatric study sponsored by Sanofi investigating unexplained enlarged spleen in patients with Gaucher disease…
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CLINICAL_TRIALS research · 2025-09-22Clinical Trial Update: A Long-term Follow-up Study of Gaucher Disease
This is a long-term follow-up clinical study on Gaucher disease that is currently recruiting at Duke University.
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CLINICAL_TRIALS research · 2025-09-12Clinical Trial Update: ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
This clinical trial update describes ScreenPlus, a comprehensive, multi-disorder newborn screening program that is currently enrolling by in…
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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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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 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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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…
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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.
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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…
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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…
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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…
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CLINICAL_TRIALS research · 2014-11-19Clinical Trial Update: Examining the Immune Response in Patients With Gaucher Disease and Hepatitis C
Clinical trial update examining the immune response in patients with Gaucher disease and hepatitis C.
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CLINICAL_TRIALS research · 2013-07-12Clinical Trial Update: Assessment Of Liver and Spleen Fibrosis in Patients With Gaucher Disease Using Fibroscan [NA]
Clinical trial update describing an observational assessment of liver and spleen fibrosis in patients with Gaucher disease using FibroScan.
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CLINICAL_TRIALS research · 2012-10-30Clinical Trial Update: Identification of Undiagnosed Gaucher Disease [NA]
A clinical trial update from Brigham and Women's Hospital on identifying undiagnosed Gaucher disease; the current status is unknown.