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Amicus Therapeutics Awarded $500,000 Grant from The Michael J. Fox Foundation for Development of Next-Generation Pharmacological Chaperone to Treat Parkinson's Disease
Date:12/8/2010

eir exciting pharmacological chaperone approach to modify the progression of Parkinson's disease," said Dr. Chesselet. "Amicus' earlier compound partially reversed the motor deficits in our alpha-synuclein overexpression mouse model, and we are eager to test the improved compounds."

John F. Crowley, Chairman and CEO of Amicus Therapeutics stated, "We appreciate MJFF's backing of our pharmacological chaperone technology and believe this grant further demonstrates the strong scientific capabilities at Amicus.  We hope that these continued discoveries will translate into therapies for people living with Parkinson's disease."

About Pharmacological Chaperones

Pharmacological chaperones are orally-available 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.  The use of pharmacological chaperones may be broadly applicable to diseases where an increase in the activity of a specific protein may provide therapeutic benefit.  

About Pharmacological Chaperones for Parkinson's Disease

Mutations in the GBA1 gene are the most common genetic risk factor known for Parkinson's disease.  Those at increased risk include individuals who inherit only one mutant copy of GBA1, while the other copy is normal.  GBA1 mutations reduce the activity of the lysosomal enzyme glucocerebrosidase (GCase), which leads to Gaucher disease when two mutant copies of the gene are inherited.  Increasing the activity of GCase in the brain with a small-molecule pharmacological chaperone may reduce the negative consequences of carrying GBA1 mutations.  Amicus and collaborators have previously  demonstrated that administration of the pharmacological chaperone AT2101 to mice that were genetically engineered to over-produce -synuclein increased the activi
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SOURCE Amicus Therapeutics
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