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The Dr. Sol J. Barer Award for
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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 biotechnology company at the forefront of advanced therapies to treat a range of devastating rare and orphan diseases. The Company has a robust development pipeline of novel, first-in-class treatments for a broad range of human genetic diseases.

Amicus is seeking global approvals of its lead product candidate, migalastat, a personalized medicine in late-stage development to treat individuals with Fabry disease on the basis of their genetic diagnosis. Zorblisa, a product candidate in late-stage development, is a potential first-to-market therapy for the chronic, rare connective tissue disorder Epidermolysis Bullosa (EB). The Company is leveraging its biologics and Chaperone-Advanced Replacement Therapy (CHARTâ„¢) platform technologies to develop next-generation ERT products for Fabry, Pompe and other lysosomal storage disorders (LSDs). Its lead biologics program is ATB200, a uniquely-engineered, next-generation Pompe ERT. Amicus is applying its CHART platform to combine ATB200 with a pharmacological chaperone to stabilize the administered enzyme in blood and potentially improve ERT tolerability and efficacy. Utilizing its biologics and CHART technologies, Amicus is also developing a next-generation Fabry ERT.
Amicus Therapeutics is committed to improving the lives of patients and families affected by rare and orphan diseases.

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