Fabry disease is a rare inherited disorder caused by a gene mutation. It narrows blood vessels, which can hurt the skin, kidneys, heart, brain, and nervous system.

Diagnosing fabry
disease can be challenging. That is because the symptoms are common and can
affect so many different parts of the body.

The symptoms include:

  • Chronic
    pain in the hands and feet;
  • Opaque
    corneas;
  • Hearing
    loss;
  • Gastrointestinal
    problems;

and many more.

Fabry disease is
confirmed with a test measuring levels of alpha-galactosidase.  

Treatment of the disease
mostly focuses on reducing the symptoms and improving the quality of life of
the patient. For instance, it can be achieved via using pain relievers and
anticlotting medications.

Contact bd@gctrials.com
to learn about GCT’s experience in conducting clinical trials in Rare Diseases,
including Fabry Disease.