Fabrazyme® (agalsidase beta)
Fabry disease is an inherited condition that affects only a few thousand people worldwide.
People with Fabry disease are missing or have insufficient amounts of the alpha-GAL enzyme, which is important in the healthy functioning of organs. The alpha-GAL enzyme is responsible for removing a fatty substance, GL-3, from cells throughout the body. Without enough of this enzyme, the GL-3 substance builds up in cells. GL-3 build-up in kidney cells may cause severe kidney problems, including kidney failure.
People with Fabry disease may experience a wide range of signs and symptoms, including severe conditions such as kidney failure, heart problems and stroke.
Patient Consent & Agreement (PCA) Form
Other Resources:
NORD - National Organization for Rare Diseases
Fabrazyme is a registered trademark of Genzyme Corporation.