Compounds for preventing and/or treating lysosomal storage disorders and/or degenerative disorders of the central nervous system
AssigneeAmicus Therapeutics, Inc.
InventorsRobert Boyd, Philip J. Rybczynski, Kamlesh Sheth
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Abstract
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.
Claims8
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.
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.
Dependencies are inferred from the claim text.