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Alzheimer's Drug Discovery Foundation Funds Amicus Therapeutics To Advance Pharmacological Chaperone Technology
Date:5/6/2010

NEW YORK, May 6 /PRNewswire-USNewswire/ -- The Alzheimer's Drug Discovery Foundation (ADDF) announced today that it has awarded a grant of $210,300 to Amicus Therapeutics (Nasdaq: FOLD; Cranbury, NJ) to evaluate small-molecule, orally delivered pharmacological chaperone compounds as a novel approach for treating Alzheimer's disease.

Amicus has discovered an apparent link between various lysosomal enzymes and accumulation of the beta-amyloid and p-tau deposits observed in the brain of Alzheimer's patients.  The ADDF's award will fund initial pre-clinical proof-of-concept studies for a specific pharmacological chaperone that targets one of these lysosomal enzymes.  

Pharmacological chaperones (PCs) are small molecules that selectively bind and stabilize target proteins to facilitate proper folding, reduce premature degradation and increase the efficiency of protein trafficking through the cell. This approach may be broadly applicable to diseases where an increase in the activity of a specific protein may provide therapeutic benefit.  

"We are honored to be selected for this grant and delighted to work with the ADDF and explore our novel approach to the treatment of Alzheimer's disease," said Dr. Brandon Wustman, Principle Investigator on the grant and Director of Exploratory Biology at Amicus Therapeutics.  "Our research at Amicus has demonstrated that studying the underlying pathology of lysosomal disorders can provide valuable insights into the mechanisms of age-related neurodegenerative diseases, and can lead to the identification of new targets for phar
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SOURCE Alzheimer's Drug Discovery Foundation
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