Treatment of glycogen storage disease type II
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, comprising administering to the individual a therapeutically effective amount of human acid α-glucosidase periodically at an administration interval, wherein the human acid α-glucosidase is a precursor of recombinant human acid α-glucosidase that has been produced in Chinese hamster ovary cell cultures, and wherein the human acid α-glucosidase has a specific enzyme activity in the range of about 1.0-3.5 μmol/min/mg protein.
2. The method of claim 1 , wherein the human acid α-glucosidase has a specific enzyme activity in the range of about 2-3.5 μmol/min/mg protein.