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Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3
Primary Objective: Evaluate the safety and pharmacokinetics of eliglustat in pediatric patients (2 to <18 years old). Secondary Objective: Evaluate the efficacy of eliglustat and quality of life in pediatric patients (2 to <18 years old).
cerezyme
alglucerase
enzyme assay
imiglucerase
gaucher disease
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- 19 Feb, 2024
- 19 locations