Glycogen Storage Disease Type II-Pompe's Disease research hub

A Study to Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated With Alglucosidase Alfa

This study is testing a new version of medicine, avalglucosidase alfa, for children aged 6 months to 17 years with a rare condition called Pompe disease. It's for children who are already on a similar medicine but aren't feeling better or are getting worse. We want to see if the new medicine is safe and works well.