DOSING REGIMENS FOR THE TREATMENT OF POMPE DISEASE

摘要

The presently disclosed subject matter provides a dosing regimen and administration schedule for the use of 1-deoxynojirimycin and enzyme replacement therapy for the treatment of Pompe disease. The presently disclosed subject matter further provides a dosing regimen and administration schedule for the use of 1-deoxynojirimycin hydrochloride and alglucosidase alfa for the treatment of Pompe disease.