IP Library Granted Patent US 7,056,712
Granted Patent B2
US 7,056,712 · App. 09/902,461 · Granted Jun 6, 2006

Treatment of glycogen storage disease type II

Assignee: Duke University
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Quick Facts
Patent No.
US 7,056,712
App. No.
09/902,461
Granted
Jun 6, 2006
Kind
B2
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 (21)

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.

Assignments (3)
MPEP 323.01(C) SUBMISSION Recorded Sep 9, 2021
From: DUKE UNIVERSITY
To: DUKE UNIVERSITY
Reel/Frame 057454/0717 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Jul 9, 2021
From: DUKE UNIVERSITY
To: SYNPAC (NORTH CAROLINA), INC.
Reel/Frame 056816/0524 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Jan 8, 2002
From: CHEN, YUAN-TSONG
To: DUKE UNIVERSITY
Reel/Frame 012446/0057 →
Continuity (2)
Provisional Application 6021923700 · Jul 18, 2000
Related Publication 20020110551A1 · Aug 15, 2002