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The Dr. Sol J. Barer Award for
Vision, Innovation and Leadership
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RareVoice Lifetime Achievement Award – Rare Disease Congressional Caucus Briefing
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Company Profile

Amicus Therapeutics (Nasdaq:FOLD) is a biopharmaceutical company at the forefront of therapies for rare and orphan diseases. The Company has a robust pipeline of novel, first-in-class treatments in development for a broad range of human genetic diseases, with a focus on delivering new benefits to individuals with lysosomal storage diseases (LSDs).

The Company is leveraging its Chaperone-Advanced Replacement Therapy (CHART™) platform to develop next-generation ERTs by identifying and co-formulating proprietary therapeutic enzymes with pharmacological chaperones. The lead CHART programs in late preclinical development are AT-B100 co-formulated with the pharmacological chaperone migalastat HCl, a next-generation ERT for Fabry disease; and AT-B200, co-formulated with the pharmacological chaperone AT2220, a next-generation ERT for Pompe disease. Amicus is also evaluating a next-generation ERT for Mucopolysaccharoidosis Type I (MPS I) in preclinical studies. Amicus retains worldwide rights to all applications of its proprietary CHART platform.

Amicus is also developing novel small molecules as monotherapy treatments for Fabry disease and Parkinson’s. Amicus owns global rights to its pharmacological chaperone, migalastat HCl, as a monotherapy for Fabry disease. In addition, the Company has entered a collaboration with Biogen Idec to discover, develop and commercialize novel small molecules for the treatment of Parkinson's disease, for further development by Biogen Idec.
Amicus Therapeutics is committed to improving the lives of patients and families affected by rare and orphan diseases.

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