Method of treating glycogen storage disease type VI
The disclosure relates, in general, to Glycogen Storage Disease Type VI and, in particular, to a method of treating Glycogen Storage Disease Type VI and to compounds and compositions suitable for use in such a method.
1. A method of treating glycogen storage disease type VI, comprising administering to a human in need thereof a composition comprising acid α-glucosidase.
2. The method of claim 1 , 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.
3. The method of claim 1 , wherein the acid α-glucosidase is a recombinant acid α-glucosidase, a precursor of recombinant acid α-glucosidase, or a combination thereof.
4. The method of claim 1 , wherein the acid α-glucosidase is administered intravenously.
5. The method of claim 1 , wherein the acid α-glucosidase is administered intrathecally.
6. The method of claim 1 , further comprising administering an immunosuppressant, an immunotherapeutic agent, or a combination thereof, concurrently or sequentially.
7. The method of claim 1 , wherein the acid α-glucosidase is administered daily.
8. The method of claim 1 , wherein the acid α-glucosidase is administered weekly.
9. The method of claim 1 , wherein the acid α-glucosidase is administered twice weekly.
10. The method of claim 1 , wherein the acid α-glucosidase is administered monthly.
11. The method of claim 1 , wherein the acid α-glucosidase is administered bi-monthly.
12. The method of claim 1 , wherein the acid α-glucosidase is administered orally, intramuscularly, intraventricularly, or a combination thereof.
13. The method of claim 3 , wherein the recombinant acid α-glucosidase or the precursor of recombinant acid α-glucosidase is produced in Chinese hamster ovary cells.
14. The method of claim 1 , wherein the acid α-glucosidase is human.