US2012058132A1PendingUtilityA1
Treatment of glycogen storage disease type II
Est. expiryJul 18, 2020(expired)· nominal 20-yr term from priority
Inventors:Yuan-Tsong Chen
A61P 43/00A61P 9/02A61P 9/00A61P 37/06A61P 9/04A61P 3/10A61P 3/00A61P 21/00A61K 38/00A61K 38/47C12Y 302/0102A61K 45/06
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Claims
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
exact text as granted — not AI-modifiedWhat is claimed is:
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.Join the waitlist — get patent alerts
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