New hope for babies battling rare, Life-Threatening muscle disease in china

This study is testing the safety and effectiveness of a drug called avalglucosidase alfa for Chinese children under 18 with infantile-onset Pompe disease, a severe genetic disorder. Participants will receive the drug by IV infusion once every two weeks for one year. The main goals are to see if the treatment is safe and if it helps children survive without a breathing machine, improves heart health, and helps them reach motor milestones like sitting or walking.