Fabry Disease : AT1001 Clinical Trials

Amicus is currently developing AT1001 (migalastat hydrochloride), an experimental, oral medication for the treatment of Fabry disease.  AT1001 acts as a pharmacological chaperone and may help to selectively stabilize and restore the three-dimensional shape, trafficking, and biological activity of α-galactosidase A (α-GAL), the enzyme that is defective in Fabry disease. Amicus is currently conducting multiple Phase 2 clinical trials to assess the effects of AT1001 in male and female patients with Fabry disease.  Enrollment in these trials was completed in March 2007.