Methods for preventing and/or treating lysosomal storage disorders
AssigneeAmicus Therapeutics, Inc.
InventorsRobert Boyd, Gary Lee
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Abstract
The present invention provides methods for preventing and/or treating lysosomal storage disorders using 5-(fluoromethyl)piperidine-3,4-diol, 5-(chloromethyl)piperidine-3,4-diol, or a pharmaceutically acceptable salt, solvate, or prodrug thereof, or any combination of two or more thereof. In particular, the present invention provides methods for preventing and/or treating Gaucher's disease.
Claims18
A method for treating a lysosomal storage disorder, which comprises administering to a patient diagnosed with at least one condition selected from the group consisting of Gaucher disease, accumulation of glucocerebroside, deficiency in glucocerebrosidase, and a mutation in a glucocerebrosidase enzyme an effective amount of a therapeutic agent selected from the group consisting of 5-(fluoromethyl)piperidine-3,4-diol, 5-(chloromethyl)piperidine-3,4-diol, a pharmaceutically acceptable salt of any of the above, a solvate of any of the above, and any combination of two or more thereof.
A method for treating Gaucher's disease in a patient diagnosed with the same, which comprises administering to the patient in need thereof an effective amount a therapeutic agent selected from the group consisting of 5-(fluoromethyl)piperidine-3,4-diol, 5-(chloromethyl)piperidine-3,4-diol, a pharmaceutically acceptable salt of any of the above, a solvate of any of the above, and any combination of two or more thereof.
A kit comprising a composition comprising an effective amount of 5-(fluoromethyl)piperidine-3,4-diol, 5-(chloromethyl)piperdine-3,4-diol, or a pharmaceutically acceptable salt, solvate, or combination thereof in a container and instructions for using the composition to treat Gaucher's disease.
Dependencies are inferred from the claim text.