Pharmacological Chaperone Technology

Amicus Therapeutics is pioneering a paradigm-shifting approach using pharmacological chaperone technology to treating a range of human genetic diseases in which crucial proteins are misfolded, thus reducing or eliminating their functionality. Amicus develops pharmacological chaperones - oral therapies that bind to and stabilize the affected proteins, restoring their shape, proper trafficking, and biological activity. This novel, next-generation approach offers the potential to improve treatment options for patients with genetic diseases through the use of pharmacological chaperone technology.