Amicus Therapeutics, Inc. is a biotechnology firm dedicated to the discovery, development, and delivery of medical treatments for rare conditions. Its primary commercial offering is Galafold, an orally administered precision medicine designed for adult patients with a confirmed diagnosis of Fabry disease, specifically those exhibiting an amenable galactosidase alpha gene variant as identified by in vitro assay data. The company's pipeline also includes several investigational therapies: AT-GAA, an innovative treatment strategy for Pompe disease; various enzyme replacement therapies aimed at Pompe diseases; and AT-GTX-502, an AAV serotype gene therapy for CLN3.