Gaucher disease type 3, Gaucher disease type 1 research hub

A 4-part, open-label, multicenter, multinational study of the safety, tolerability, pharmacokinetics, pharmacodynamic, and exploratory efficacy of venglustat in combination with Cerezyme in adult patients with Gaucher disease Type 3 with venglustat monotherapy extension

This study is looking at a new medicine called venglustat for adults with Gaucher disease, particularly Type 3. It wants to see if the new medicine is safe, how the body handles it, and if it might help. It's also comparing it with an existing treatment.