Amicus Therapeutics

Amicus Therapeutics (NASDAQ: FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing, and delivering high-quality medicines for people living with rare metabolic diseases. The company has a novel, first-in-class pipeline of small molecule products known as pharmacological chaperones for the treatment of lysosomal storage disorders (LSDs). These chaperones may offer a dual therapeutic approach for Fabry, Pompe, Gaucher, and other LSDs. As orally administered monotherapy drugs, pharmacological chaperones are designed to bind, stabilize, and enhance the activity of a patient’s own misfolded enzyme. When used in combination with enzyme replacement therapy (ERT), pharmacological chaperones may improve the uptake of the infused enzyme and potentially enhance ERT outcomes.