Open-label bridging study of Myozyme in pediatric late-onset Pompe disease

Children and adolescents aged 5 to less than 18 years with confirmed Pompe disease, documented by GAA mutation analysis or deficient endogenous GAA activity, demonstrable muscle weakness, reproducible sitting FVC testing, ambulatory ability of at least 10 meters, and ability to perform muscle function testing. Patients were excluded for prior GAA enzyme replacement therapy, recent investigational treatment, invasive ventilation, daytime upright non-invasive ventilation, pregnancy, or inability to comply with the protocol.