Gaucher's disease type I research hub

Open label, two cohort (with and without imiglucerase), multicenter study to evaluate pharmacokinetics, safety, and efficacy of eliglustat in pediatric patients with Gaucher disease type 1 and type 3

This study is looking at a new medicine called eliglustat for children with Gaucher disease types 1 and 3. It wants to understand how the medicine works in their bodies, if it's safe, and if it helps improve their symptoms, sometimes alongside another treatment called Cerezyme.