Cerezyme 400 Units Powder for concentrate for solution for infusion clinical trials

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.