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 diagnosed with the same, which comprises administering to the patient in need thereof an effective amount of (3R,4R,5S)-5-(difluoromethyl)piperidine-3,4-diol or a pharmaceutically acceptable salt thereof.
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 includes a therapeutic agent selected from imiglucerase and 1,5-(butylimino)-1,5-dideoxy-D-glucitol.
4. A method for treating type 1 Gaucher's disease in a patient diagnosed with the same, which comprises administering to the patient in need thereof an effective amount of (3R,4R,5S)-5-(1-fluoroethyl)piperidine-3,4-diol or a pharmaceutically acceptable salt thereof.
5. The method of claim 4 , further comprising administering an effective amount of at least one other therapeutic agent.
6. The method of claim 5 , wherein the at least one other therapeutic agent includes a therapeutic agent selected from imiglucerase and 1,5-(butylimino)-1,5-dideoxy-D-glucitol.
7. A method for treating type 1 Gaucher's disease in a patient diagnosed with the same, which comprises administering to the patient in need thereof an effective amount of (3R,4R,5S)-5-((R)-1-fluoropropyl)piperidine-3,4-diol or a pharmaceutically acceptable salt thereof.
8. The method of claim 7 , further comprising administering an effective amount of at least one other therapeutic agent.
9. The method of claim 8 , wherein the at least one other therapeutic agent includes a therapeutic agent selected from imiglucerase and 1,5-(butylimino)-1,5-dideoxy-D-glucitol.
10. The method of claim 7 , wherein an effective amount of (3R,4R,5S)-5-((R)-1-fluoropropyl)piperidine-3,4-diol hydrochloride is administered to the patient.
11. The method of claim 10 , further comprising administering an effective amount of at least one other therapeutic agent.
12. The method of claim 11 , wherein the at least one other therapeutic agent includes a therapeutic agent selected from imiglucerase and 1,5-(butylimino)-1,5-dideoxy-D-glucitol.