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.

Long Term Follow-up Study of Type-1 Gaucher Subjects Post FLT201 Dose (GALILEO-2)

This study is checking on the long-term health of people with Type 1 Gaucher disease who previously received a treatment called FLT201 in an earlier trial. We'll follow them for five years to see how they're doing.

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

A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)

This study is testing a new medicine, LY3884961, for adults with Gaucher disease, type 1. It aims to find the right dose and see how safe and effective it is for symptoms affecting the body, but not the brain.