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Overview |
FAQs |
Lysosomal storage disorders
Fabry disease is a rare inherited disorder characterized by a deficiency of the alpha-GAL enzyme, which normally removes a fatty substance (GL-3) from cells throughout the body. Buildup of GL-3 in kidney cells can eventually cause kidney failure. Severe cases may also result in heart problems and stroke.
Standard treatment is with Fabrazyme®.
Fabrazyme is a registered trademark of Genzyme Corporation.
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