Amicus Therapeutics Inc (Amicus Therapeutics) develops novel, oral therapeutics for the treatment of rare genetic diseases known as lysosomal storage disorders (LSDs). The company specializes in research, development and marketing of small molecule drugs and orally administered monotherapy for the treatment of Fabry disease and other lysosomal storage diseases such as Pompe disease.

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Amicus Therapeutics conducts R&D to advance its product candidates and explore new uses for its pharmacological chaperone technology. Thorough collaboration with Perelman School of Medicine at the University of Pennsylvania, the company strives to discover and develop novel gene therapies for the treatment of Pompe disease, cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD), Fabry disease, and an undisclosed rare metabolic disorder. It has a laboratory in Philadelphia, Pennsylvania, the US. In FY2022, the company incurred R&D expenses of US$276.6 million, which as a percentage of revenue stood at 84.01%.