Amicus Therapeutics Inc incorporated under the laws of the State of Delaware on February 4, 2002. The Company is a Biopharmaceutical Company focused on the discovery, development and commercialization of next-generation medicines for a range of rare and orphan diseases, with a focus on improved therapies for lysosomal storage disorders (LSDs). IT is focused on developing pharmacological chaperones as monotherapy agents (to be used without ERT) and its Chaperone- Advanced Replacement Therapy, or CHART platform of pharmacological chaperones in combination with ERT. Chaperone- Advanced Replacement Therapy, or CHART, platform has been used to develop our next-generation ERTs by co-formulating therapeutic enzymes with our proprietary pharmacological chaperones. It is also developing migalastat HCl as a monotherapy in two Phase 3 global registration studies ("Study 011" and "Study 012") for Fabry patients with genetic mutations that were amenable to this pharmacological chaperone in a cell-based assay. Study 011 is a 24-month study consisting of a 6-month double-blind, placebo-controlled treatment period (Stage 1); a 6-month open-label follow-up period (Stage 2); and a 12-month open-label extension phase. The Company currently operates in one business segment focusing on the development and commercialization of small molecule, orally administered therapies to treat a range of human genetic diseases. Its competitors include pharmaceutical and biotechnology companies in the U.S. and abroad that have approved therapies or therapies in development for lysosomal storage disorders within core programs. The Company owns certain trademarks in the U.S. and/or abroad, including A AMICUS THERAPEUTICS & design, AMICUS THERAPEUTICS and CHART.
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