摘要:
The present application provides for compositions comprising α-galactosidase A in combination with an active site-specific chaperone for the α-galactosidase A, and methods for treating Fabry disease in a subject in need thereof, that includes a method of administering to the subject such compositions. The present application also provides methods for increasing the in vitro and in vivo stability of an α-galactosidase A enzyme formulation.
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.
摘要:
The present application provides for compositions comprising high concentrations of acid a-glucosidase in combination with an active site-specific chaperone for the acid α-glucosidase, and methods for treating Pompe disease in a subject in need thereof, that includes a method of administering to the subject such compositions. The present application also provides methods for increasing the in vitro and in vivo stability of an acid α-glucosidase enzyme formulation.
摘要:
The presently disclosed subject matter provides a dosing regimen and administration schedule for the use of 1-deoxygalactonojirimycin and enzyme replacement therapy for the treatment of Fabry disease. The presently disclosed subject matter further provides a dosing regimen and administration schedule for the use of migalastat hydrochloride and agalsidase for the treatment of Fabry disease.