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Gaucher Disease Type 1
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Gaucher Disease Type 1 trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
See which Gaucher Disease Type 1 trials may be worth asking aboutNorth America
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This study is a Phase 3, non-randomized, multicenter, efficacy and safety study in adult patients with Gaucher disease Type 1, on stable treatment with enzyme r…
Study J3Z-MC-OJAE is a Phase 1/2, multicenter, open-label, dose-finding study of LY3884961 evaluating the safety and tolerability in adults with peripheral mani…
Objective of the trial. To define a sub-population which is at increased risk of developing Parkinson, beyond the fact of carrying Gaucher; in this sub-populati…
The objective of this study is to evaluate oxidative stress and/or inflammation in patients with Gaucher disease type I using a series of biomarkers and correla…
Gaucher disease (GD) is caused by mutations in the GBA1 gene, which leads to a lack or reduction of GCase activity. The consequences of this deficiency are gene…
This is a data collection study that will examine the general diagnostic and treatment data associated with the reduced-intensity chemotherapy-based regimen pai…
Primary Objective: Evaluated the safety and pharmacokinetics of eliglustat in pediatric participants (≥2 to \<18 years old). Secondary Objective: Evaluated t…
Part 1: Biomarker evaluation/screening phase Primary Objectives: * Evaluate cerebrospinal fluid (CSF) biomarkers in adult Gaucher disease Type 3 (GD3) partici…