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 . At the screen, please check the box that says “Check if you have no code” to proceed to the RSVP.