Treatment of Glycogen Storage Disease Type II

Abstract

Methods of treating glycogen storage disease type II, by administering acid α-glucosidase, are described, as are compositions for use in treatment of glycogen storage disease type II.

1 . A method of treating glycogen storage disease type II in a human individual having glycogen storage disease type II (GSD-II), comprising: administering to the individual a therapeutically effective amount of human acid α-glucosidase (hGAA) periodically at an administration interval, wherein the hGAA is a precursor of recombinant hGAA that has been produced in Chinese hamster ovary cell cultures, and wherein the therapeutically effective amount of hGAA is varied over time depending on the medical needs of the individual. 
     
     
         2 . The method of  claim 1 , wherein the therapeutically effective amount of hGAA is increased when the individual is in times of physical illness with GSD-II. 
     
     
         3 . The method of  claim 1 , wherein the therapeutically effective amount of hGAA is increased when the individual is in times of stress. 
     
     
         4 . The method of  claim 1 , wherein the therapeutically effective amount of hGAA is increased when symptoms of GSD-II worsen. 
     
     
         5 . The method of  claim 1 , wherein the therapeutically effective amount of hGAA is increased when the individual develops antibodies to the hGAA. 
     
     
         6 . The method of  claim 1 , wherein the therapeutically effective amount of hGAA is increased when the individual develops increased levels of antibodies to the hGAA.