Treatment of glycogen storage disease type II
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.
1. A method of treating glycogen storage disease type II in a human in need of such treatment comprising:
i) determining if said human is cross-reactive-immunological-material negative for endogenous acid α-glucosidase (CRIM-negative), and
ii) to a human determined in step (i) to be CRIM-negative, administering recombinant human acid α-glucosidase from Chinese hamster ovary cell culture (CHO GAA) in an amount sufficient to effect said treatment, in conjunction with an immunosuppressive or immunotherapeutic regimen.
2. The method according to claim 1 , wherein said immunosuppressive or immunotherapeutic regimen is administered prior to any administration of said CHO GAA.
3. A method of treating glycogen storage disease type II in a CRIM-negative human in need of such treatment comprising administering CHO GAA to the CRIM-negative human in an amount sufficient to effect said treatment in conjunction with an immunosuppressive or immunotherapeutic regimen.
4. The method according to claim 3 , wherein said immunosuppressive or immunotherapeutic regimen is administered prior to any administration of said CHO GAA.
5. The method according to claim 1 or 3 , wherein the glycogen storage disease type II is infantile glycogen storage disease type II, juvenile glycogen storage disease type II, or adult-onset glycogen storage disease type II.
6. The method according to claim 5 , wherein the glycogen storage disease type II is infantile glycogen storage disease type II.
7. The method according to claim 1 or 3 , wherein less than 15 mg of said CHO GAA per kilogram of body weight is administered.
8. The method according to claim 7 , wherein 1 to 10 mg of said CHO GAA per kilogram of body weight is administered.
9. The method according to claim 1 or 3 , wherein said CHO GAA is the precursor form of human acid α-glucosidase.
10. The method according to claim 1 or 3 , wherein said CHO GAA is administered at a regular interval.
11. The method according to claim 10 , wherein said CHO GAA is administered monthly, bimonthly, weekly, twice weekly or daily.
12. The method according to claim 11 , wherein said CHO GAA is administered bimonthly.
13. The method according to claim 1 or 3 , wherein said CHO GAA is administered intravenously, intramuscularly, intrathecally or intraventricularly.
14. The method according to claim 1 or 3 , wherein said method comprises treating cardiomyopathy associated with glycogen storage disease type II in a human in need of such treatment.
15. A method of treating glycogen storage disease type II in a CRIM-negative human in need of such treatment comprising administering CHO GAA bimonthly to the human in an amount sufficient to effect said treatment, in conjunction with an immunosuppressive or immunotherapeutic regimen, wherein said CHO GAA is the precursor form of human acid α-glucosidase.
16. The method according to claim 15 , wherein said immunosuppressive or immunotherapeutic regimen is administered prior to any administration of said CHO GAA.