Medication Use in Fabry Disease

Fabry disease is a rare inherited disorder of glycosphingolipid (fat) metabolism resulting from the absent or markedly deficient activity of the lysosomal enzyme, α-galactosidase A (α-Gal A). This research is for patients who have been diagnosed with Fabry disease and aims to learn about how patients impacted by Fabry disease who have a genetic mutation that is amenable to taking a chaperone drug make therapy choices.

If you are a patient diagnosed with Fabry disease and you are interested in learning more or participating in the study, please click here. At the screen, please check the box that says “Check if you have no code” to proceed to the RSVP.