Methods for treating glycogen storage disease type IV usng lysosomal acid alpha-glucosidase
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.
1. A method for treating glycogen storage disease type IV comprising administering a lysosomal acid α-glucosidase.
2. The method of claim 1 , wherein the amount of the lysosomal acid α-glucosidase administered is less than about 40 mg of acid α-glucosidase per kilogram of body weight.
3. The method of claim 1 , wherein the lysosomal acid α-glucosidase is recombinant human lysosomal acid α-glucosidase or a precursor of recombinant human lysosomal acid α-glucosidase.
4. The method of claim 3 , wherein the recombinant human lysosomal acid α-glucosidase or the precursor of recombinant human lysosomal acid α-glucosidase is produced in Chinese hamster ovary cells.
5. The method of claim 1 , wherein the lysosomal acid α-glucosidase is administered monthly.
6. The method of claim 1 , wherein the lysosomal acid α-glucosidase is administered intravenously.
7. The method of claim 1 , wherein the lysosomal acid α-glucosidase is administered in conjunction with, or subsequent to, administration of an immunosuppressant.
8. The method of claim 1 , wherein the lysosomal acid α-glucosidase is administered orally, intramuscularly, or intraventricularly.
9. The method of claim 1 , wherein the lysosomal acid α-glucosidase is administered bi-monthly.
10. The method of claim 1 , wherein the lysosomal acid α-glucosidase is administered weekly.
11. The method of claim 1 , wherein the lysosomal acid α-glucosidase is administered twice weekly.
12. The method of claim 1 , wherein the lysosomal acid α-glucosidase is administered daily.
13. The method of claim 1 , wherein the lysosomal acid α-glucosidase is administered by two or more of orally, intravenously, intramuscularly, and intraventricularly.
14. The method of claim 1 , wherein the lysosomal acid α-glucosidase is human.
15. The method of claim 1 , wherein the lysosomal acid α-glucosidase is administered intrathecally.