HIGH CONCENTRATION ALPHA-GLUCOSIDASE COMPOSITIONS FOR THE TREATMENT OF POMPE DISEASE

摘要

The present application relates to Pompe disease comprising a composition comprising a high concentration of acidic α-glucosidase in combination with an active site-specific chaperone for acidic α-glucosidase, and a method of administering the composition to a subject. A method of treating Pompe disease in a subject in need of treatment is provided. The present application also provides a method of increasing the in vitro and in vivo stability of an acidic α-glucosidase enzyme formulation.