Method for the treatment of neurological disorders by enhancing the activity of beta-glucocerebrosidase

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United States of America Patent

PATENT NO 9119845
APP PUB NO 20110052613A1
SERIAL NO

12941468

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Abstract

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Provided is a method of increasing the stability of wild-type β-glucocerebrosidase. Also provided are methods of treating and/or preventing an individual having a neurological disease in which increased expression or activity of β-glucocerebrosidase in the central nervous system would be beneficial. This method includes administering an effective amount of a pharmacologic chaperone for β-glucocerebrosidase, with the proviso that the individual does not have a mutation in the gene encoding β-glucocerebrosidase. Further provided are β-glucocerebrosidase inhibitors which have been identified as specific pharmacologic chaperones and which have been shown to increase activity of β-glucocerebrosidase in vivo in the central nervous system.

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Patent Owner(s)

Patent OwnerAddress
AMICUS THERAPEAUTICS INCCRANBURY NJ

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Inventor(s)

Inventor Name Address # of filed Patents Total Citations
Wustman, Brandon Alan San Diego, US 11 141

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