Amicus Therapeutics, Inc. is a biopharmaceutical company. The Company is focused on the discovery and development of therapies to treat a range of rare and orphan diseases. Its lead product candidate, migalastat HCl (Galafold), is an orally administered small molecule pharmacological chaperone for the treatment of Fabry disease, which is a type of lysosomal storage disorder (LSD). It has completed two Phase III global registration studies (Study 011 and Study 012) of migalastat monotherapy. It is also engaged in Phase III clinical development of a topical cream, SD-101, for the treatment of the genetic connective tissue disorder Epidermolysis Bullosa. It has also initiated a clinical study in patients with Pompe disease, a LSD to investigate its treatment paradigm that consists of ATB200, which is an engineered recombinant human acid alpha-glucosidase enzyme with an optimized carbohydrate structure to enhance uptake, co-administered with AT2221, to improve activity and stability.