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.
As of June 2 at 8:00 am CT, the study is now closed, thank you to all who have participated!
If you would like to sign up for future research, please click here and we will contact you as opportunities become available.