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Amicus Therapeutics, Inc. is a biotechnology company. The Company is engaged in the discovery, development and commercialization of a set of treatments for patients living with devastating rare and orphan diseases. Its lead product, migalastat HCl is a small molecule that can be used as a monotherapy and in combination with enzyme replacement therapy (ERT) for Fabry disease. Its pipeline also includes SD-101, which is a product candidate in late-stage development, as a potential first-to-market therapy for the chronic, rare connective tissue disorder Epidermolysis Bullosa (EB). It is also leveraging its Chaperone-Advanced Replacement Therapy (CHART) platform technologies to develop ERT products for Pompe disease, Fabry disease, and potentially other lysosomal storage disorders (LSDs). The Company is also investigating preclinical and discovery programs in other rare and devastating diseases, including cyclin-dependent kinase-like 5 (CDKL5) deficiency.