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 was produced in chinese hamster ovary cell cultures.
2. The method of claim 1 , wherein the glycogen storage disease type II is infantile glycogen storage disease type II.
3. The method of claim 1 , wherein the glycogen storage disease type II is juvenile glycogen storage disease type II.
4. The method of claim 1 , wherein the glycogen storage disease type II is adult-onset glycogen storage disease type II.
5. The method of claim 1 , wherein the therapeutically effective amount of human acid α-glucosidase is less than about 15 mg of acid α-glucosidase per kilogram of body weight of the individual.
6. The method of claim 5 , wherein the therapeutically effective amount of human acid α-glucosidase is about 1–10 mg of acid α-glucosidase per kilogram of body weight of the individual.
7. The method of claim 5 , wherein the therapeutically effective amount of human acid α-glucosidase is about 5 mg of acid α-glucosidase per kilogram of body weight of the individual.
8. The method of claim 1 , wherein the human acid α-glucosidase is recombinant human acid α-glucosidase that has been produced in chinese hamster ovary cell cultures.
9. The method of claim 1 , 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.
10. The method of claim 1 , wherein the administration interval is monthly.
11. The method of claim 1 , wherein the administration interval is bimonthly.
12. The method of claim 1 , wherein the administration interval is weekly.
13. The method of claim 1 , wherein the administration interval is twice weekly.
14. The method of claim 1 , wherein the administration interval is daily.
15. The method of claim 1 , wherein the human acid α-glucosidase is administered intravenously.
16. The method of claim 1 , wherein the human acid α-glucosidase is administered intramuscularly.
17. The method of claim 1 , wherein the human acid α-glucosidase is administered intrathecally or intraventricularly.
18. The method of claim 1 , wherein the human acid α-glucosidase is administered in conjunction with an immunosuppressant.
19. The method of claim 18 , wherein the immunosuppressant is administered prior to any administration of human acid α-glucosidase to the individual.
20. A method of treating cardiomyopathy associated with glycogen storage disease type II in an 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 was produced in chinese hamster ovary cell culture.
21. The method of claim 1 , wherein the administration interval is varied over time.