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Found 1 clinical trials

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Multi-country trial
Phase 3
Recruiting in 10s
Interventional Study
all
Genetic Disease
Recruiting

Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3

This is a parallel arm, Phase 3, double-blind, double-dummy, active-comparator, 2 arm study to evaluate the efficacy and safety of daily oral venglustat versus intravenous Cerezyme infusions

deficiency

cerezyme

platelet count

imiglucerase

replacement therapy

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03 Dec, 2024
18 locations

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