Compositions for preventing and/or treating lysosomal storage disorders
View Patent ↗The present invention provides novel compositions as well as methods for preventing and/or treating lysosomal storage disorders. In particular, the present invention provides methods for preventing and/or treating Gaucher's disease.
1. A method for treating type 1 Gaucher's disease in a patient in need of such treatment, which comprises administering to the patient an effective amount of a compound of Formula III:
wherein:
R 1 is C(R 2 )(R 3 )(R 4 );
R 2 is hydrogen or fluorine;
R 3 is fluorine;
R 4 is fluorine, C 1-4 alkyl, or phenyl;
R 7 is —OH; and
R 8 is hydrogen.
2. The method of claim 1 , further comprising administering an effective amount of at least one other therapeutic agent.
3. The method of claim 2 , wherein the at least one other therapeutic agent is imiglucerase or 1,5-(butylimino)-1,5-dideoxy-D-glucitol.
4. The method of claim 1 , wherein R 2 is hydrogen.
5. The method of claim 1 , wherein R 2 is fluorine.
6. The method of claim 1 , wherein R 4 is fluorine.
7. The method of claim 1 , wherein R 4 is C 1-4 alkyl.
8. The method of claim 1 , wherein R 4 is phenyl.