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a world of hope for families and
individuals with Gaucher Disease

Amicus Therapeutics

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Who We Are
Founded in 2002, Amicus Therapeutics is pioneering a paradigm-shifting approach to treating a range of human genetic diseases in which crucial proteins are misfolded, thus reducing or eliminating their functionality. Amicus develops pharmacological chaperones - oral therapies that bind to and stabilize the affected proteins, restoring their shape, proper trafficking, and biological activity.

This novel, next-generation approach offers the potential to improve treatment options for patients with genetic diseases. Amicus Therapeutics is a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of a new class of small molecule, orally-administered drugs to treat a range of human genetic diseases.

Amicus' innovative therapies apply to conditions in which crucial proteins are defective as a result of improper folding.

Instead of trying to replace these complex proteins, Amicus' approach uses pharmacological chaperones to selectively bind to the misfolded target protein, increase stability, and help the protein fold into its proper three-dimensional shape.

This allows the protein to be trafficked to the specific location in the cell, where it performs its intended biological function. Pharmacological chaperone technology represents a novel, next-generation approach to the management of human genetic diseases and offers the potential to improve treatment options for patients. (Information from
www.amicustherapeutics.com).

Contact information:
Amicus Therapeutics, Inc.
6 Cedar Brook Drive
Cranbury, NJ 08512
Tel: 609-662-2000
Fax: 609-662-2001

General Information:
info@amicustherapeutics.com
Wwebsite: www.amicustherapeutics.com

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National Gaucher Foundation

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800-504-3189