New hope for babies with rare muscle disease: enzyme therapy trial launches in china

This study tests a drug called avalglucosidase alfa in 13 Chinese children with infantile-onset Pompe disease, a rare genetic condition that weakens the heart and muscles. The treatment is given as an IV infusion every two weeks for 52 weeks. Researchers will check if it helps children live longer without needing a breathing machine and improves heart function. The goal is to see if this enzyme replacement therapy is safe and effective for this group.