Compounds for preventing and/or treating lysosomal storage disorders and/or degenerative disorders of the central nervous system
US-9464050-B2 · Oct 11, 2016 · US
US10035766B2 · US · B2
| Field | Value |
|---|---|
| Publication number | US-10035766-B2 |
| Application number | US-201314083634-A |
| Country | US |
| Kind code | B2 |
| Filing date | Nov 19, 2013 |
| Priority date | Oct 19, 2009 |
| Publication date | Jul 31, 2018 |
| Grant date | Jul 31, 2018 |
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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.
Opening claim text (preview).
What is claimed: 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.
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