Cerdelga shows benefits in children with Gaucher disease type 1: Study
Treatment with Cerdelga (eliglustat), a substrate reduction therapy approved in the U.S. for adults with type 1 Gaucher disease, was safe and showed therapeutic potential in children with the condition, a real-world study finds. “This case series highlights real-world experience with [Cerdelga] in pediatric [Gaucher type 1] patients,…