Compounds for preventing and/or treating lysosomal storage disorders and/or degenerative disorders of the central nervous system
Described are novel salts of the compound (3R,4R,5S)-5-(difluoromethyl) piperidine-3,4-diol, as well as methods of using the same for preventing and/or treating lysosomal storage disorders and/or degenerative disorders of the central nervous system. In particular, the present invention provides methods for preventing and/or treating Gaucher's disease and/or Parkinson's disease.
1. A method for treating Parkinson's disease in a patient at risk for developing or diagnosed with the same, which comprises administering to the patient an effective amount of a crystalline form of (3R,4R,5S)-5-(difluoromethyl)piperidine-3,4-diol•fumarate (1:1), wherein the crystalline form has an X-ray diffraction pattern comprising characteristic peaks at:
2-Theta (Degrees)
11.70 ± 0.2
15.70 ± 0.2
20.78 ± 0.2
21.46 ± 0.2
27.74 ± 0.2.
2. The method of claim 1 , wherein the crystalline form has an X-ray diffraction pattern comprising characteristic peaks at:
2-Theta (Degrees)
11.70 ± 0.2
14.66 ± 0.2
15.70 ± 0.2
19.34 ± 0.2
20.78 ± 0.2
21.46 ± 0.2
22.22 ± 0.2
26.26 ± 0.2
26.90 ± 0.2
27.74 ± 0.2.
3. The method of claim 1 , wherein the crystalline form has an X-ray diffraction pattern of FIG. 1 .
4. The method of claim 1 , wherein the crystalline form is administered as part of a pharmaceutical composition comprising a pharmaceutically acceptable carrier.
5. A method for treating Gaucher's disease in a patient at risk for developing or diagnosed with the same, which comprises administering to the patient an effective amount of a crystalline form of (3R,4R,5S)-5-(difluoromethyl)piperidine-3,4-diol.fumarate (1:1), wherein the crystalline form has an X-ray diffraction pattern comprising characteristic peaks at:
2-Theta (Degrees)
11.70 ± 0.2
15.70 ± 0.2
20.78 ± 0.2
21.46 ± 0.2
27.74 ± 0.2.
6. The method of claim 5 , wherein the crystalline form has an X-ray diffraction pattern comprising characteristic peaks at:
2-Theta (Degrees)
11.70 ± 0.2
14.66 ± 0.2
15.70 ± 0.2
19.34 ± 0.2
20.78 ± 0.2
21.46 ± 0.2
22.22 ± 0.2
26.26 ± 0.2
26.90 ± 0.2
27.74 ± 0.2.
7. The method of claim 5 , wherein the crystalline form has an X-ray diffraction pattern of FIG. 1 .
8. The method of claim 5 , wherein the crystalline form is administered as part of a pharmaceutical composition comprising a pharmaceutically acceptable carrier.