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.

主权项

1. A method of treating Pompe disease in a subject, the method comprising administering from about 25 mg to about 1000 mg of 1-deoxynojirimycin and an effective amount of acid α-glucosidase enzyme replacement therapy to a patient in need thereof.