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Last Updated: December 24, 2024

Claims for Patent: 10,035,766


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Summary for Patent: 10,035,766
Title:Compositions for preventing and/or treating lysosomal storage disorders
Abstract: 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.
Inventor(s): Boyd; Robert (Horsham, PA), Lee; Gary (West Windsor, NJ), Rybczynski; Philip (Branchburg, NJ)
Assignee: Amicus Therapeutics, Inc. (Cranbury, NJ)
Application Number:14/083,634
Patent Claims: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: ##STR00035## wherein: R.sup.1 is C(R.sup.2)(R.sup.3)(R.sup.4); R.sup.2 is hydrogen or fluorine; R.sup.3 is fluorine; R.sup.4 is fluorine, C.sub.1-4 alkyl, or phenyl; R.sup.7 is --OH; and R.sup.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.sup.2 is hydrogen.

5. The method of claim 1, wherein R.sup.2 is fluorine.

6. The method of claim 1, wherein R.sup.4 is fluorine.

7. The method of claim 1, wherein R.sup.4 is C.sub.1-4 alkyl.

8. The method of claim 1, wherein R.sup.4 is phenyl.

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