A phase 3, multicenter, multinational, randomized, doubleblind, double-dummy, active-comparator study to evaluate the efficacy and safety of venglustat in adult and pediatric patients with Gaucher disease Type 3 (GD3) who have reached therapeutic goals with Enzyme Replacement Therapy (ERT)

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Balcı M. C. (Executive), Gökçay G. F., Karaca M., Aktaş Ü.

Industrial Organizations of Other Countries Supported Project, 2024 - 2026

• Project Type: Industrial Organizations of Other Countries Supported Project
• Begin Date: June 2024
• End Date: June 2026

Project Abstract

A phase 3, multicenter, multinational, randomized, doubleblind, double-dummy, active-comparator study to evaluate the efficacy and safety of venglustat in adult and pediatric patients with Gaucher disease Type 3 (GD3) who have reached therapeutic goals with Enzyme Replacement Therapy (ERT)