Gaucher Disease Type 1 research hub

Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension

This study is looking at a new medication, venglustat, for adults with a rare genetic condition called Gaucher disease, specifically types 1 and 3. Researchers want to see if venglustat, alone or with another drug called Cerezyme, is safe and helps improve symptoms.

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