Overview

Amicus Therapeutics, Inc. (Amicus) is a biopharmaceutical company focused on the discovery, development and commercialization of orally administered, small molecule drugs known as pharmacological chaperones for treating a range of diseases, including lysosomal storage diseases and diseases of neurodegeneration. It is focused on the development of pharmacological chaperone monotherapy programs and pharmacological chaperones in combination with enzyme replacement therapy (ERT). Its approach to the treatment of human genetic diseases consists of using pharmacological chaperones that selectively bind to the target protein. Amicus is also conducting preclinical studies on the use of its pharmacological chaperone technology to treat Parkinson s disease, with an initial focus on Parkinson s disease patients who are also Gaucher disease carriers.

Endorsements