IP Library › Patent Application 13064556
Patent Application
App. No. 13/064,556

Treatment of glycogen storage disease type II

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Quick Facts
Patent No.
US None
App. No.
13/064,556
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.

Claims (22)

1 . A method of treating glycogen storage disease type II in an individual, comprising administering to the individual a therapeutically effective amount of human acid α-glucosidase at a regular interval.

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.

9 . The method of claim 1 , wherein the human acid α-glucosidase is a precursor of recombinant human acid α-glucosidase.

10 . The method of claim 9 , wherein the recombinant human acid α-glucosidase is produced in Chinese hamster ovary cells.

11 . The method of claim 1 , wherein the regular interval is monthly.

12 . The method of claim 1 , wherein the regular interval is bimonthly.

13 . The method of claim 1 , wherein the regular interval is weekly.

14 . The method of claim 1 , wherein the regular interval is twice weekly.

15 . The method of claim 1 , wherein the regular interval is daily.

16 . The method of claim 1 , wherein the human acid α-glucosidase is administered intravenously.

17 . The method of claim 1 , wherein the human acid α-glucosidase is administered intramuscularly.

18 . The method of claim 1 , wherein the human acid α-glucosidase is administered intrathecally or intraventricularly.

19 . The method of claim 1 , wherein the human acid α-glucosidase is administered in conjunction with an immunosuppressant.

20 . The method of claim 19 , wherein the immunosuppressant is administered prior to any administration of human acid α-glucosidase to the individual.

21 . A method of treating cardiomyopathy associated with glycogen storage disease type II in an individual, comprising administering to the individual a therapeutically effective amount of human acid α-glucosidase at a regular interval.

22 . A pharmaceutical composition comprising human acid α-glucosidase in a container with a label containing instructions for administration of the composition for treatment of glycogen storage disease type II.