Glycogen Storage Disease Type II Infantile Onset research hub

A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18

This study looks at a new treatment called cipaglucosidase alfa/miglustat for children with a rare genetic condition called Infantile Onset Pompe Disease (IOPD). It checks if the treatment is safe and works, and how the body handles it. It includes children who have and haven't had previous treatments.