US2022106579A1PendingUtilityA1

Method of Treating Glycogen Storage Disease

Assignee: UNIV DUKEPriority: Jul 8, 2008Filed: Dec 13, 2021Published: Apr 7, 2022
Est. expiryJul 8, 2028(~2 yrs left)· nominal 20-yr term from priority
A61P 21/00A61K 38/47A61P 1/16C12Y 302/0102A61P 1/00C12N 9/2408A61P 3/00A61P 37/06
74
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Claims

Abstract

The disclosure relates, in general, to Glycogen Storage Disease and, in particular, to a method of treating Glycogen Storage Disease and to compounds and compositions suitable for use in such a method.

Claims

exact text as granted — not AI-modified
1 - 18 . (canceled) 
     
     
         19 . A method of treating glycogen storage disease type XI or cardiac glycogenosis, the method comprising:
 administering to a human in need thereof a composition comprising acid α-glucosidase.   
     
     
         20 . The method of  claim 19 , wherein the amount of the acid α-glucosidase administered is from about 1 mg to about 40 mg of acid α-glucosidase per kilogram of body weight. 
     
     
         21 . The method of  claim 19 , wherein the acid α-glucosidase is a recombinant acid α-glucosidase, a precursor of recombinant acid α-glucosidase, or a combination thereof. 
     
     
         22 . The method of  claim 19 , wherein the acid α-glucosidase is administered intravenously. 
     
     
         23 . The method of  claim 19 , wherein the acid α-glucosidase is administered intrathecally. 
     
     
         24 . The method of  claim 19 , further comprising administering an immunosuppressant, an immunotherapeutic agent, or a combination thereof, concurrently, or sequentially. 
     
     
         25 . The method of  claim 19 , wherein the acid α-glucosidase is administered daily. 
     
     
         26 . The method of  claim 19 , wherein the acid α-glucosidase is administered weekly. 
     
     
         27 . The method of  claim 19 , wherein the acid α-glucosidase is administered twice weekly. 
     
     
         28 . The method of  claim 19 , wherein the acid α-glucosidase is administered monthly. 
     
     
         29 . The method of  claim 19 , wherein the acid α-glucosidase is administered bi-monthly. 
     
     
         30 . The method of  claim 19 , wherein the acid α-glucosidase is administered orally, intramuscularly, intraventricularly, or a combination thereof. 
     
     
         31 . The method of  claim 21 , wherein the recombinant acid α-glucosidase or the precursor of recombinant acid α-glucosidase is produced in Chinese hamster ovary cells. 
     
     
         32 . The method of  claim 19 , wherein the acid α-glucosidase is human. 
     
     
         33 . A method of treating an individual who has been diagnosed as having glycogen storage disease type XI or cardiac glycogenosis, the method comprising:
 administering by injection to an individual following diagnosis of glycogen storage disease type XI or cardiac glycogenosis of human acid α-glucosidase at a regular interval sufficient to effect the treatment.   
     
     
         34 . The method of  claim 33 , wherein the regular interval is daily. 
     
     
         35 . The method of  claim 33 , wherein the regular interval is weekly. 
     
     
         36 . The method of  claim 33 , wherein the regular interval is twice weekly. 
     
     
         37 . The method of  claim 33 , wherein the regular interval is monthly. 
     
     
         38 . The method of  claim 33 , wherein the regular interval is bi-monthly.

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