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TV Health Reports: Air Date: September 15, 2002
Fabry's Disease
University of Iowa Children's Hospital at the University of Iowa Hospitals & Clinics
in Iowa City is one of 20 medical centers taking part in a major international
study. Researchers are looking at a new treatment for Fabry's Disease --
a rare genetic disorder that begins in childhood and often leads to death
by age 30.
Fabry's disease is an inherited condition that affects about one in 40
thousand males. The body is unable to break down certain naturally occuring
lipids. Thomas Loew, M.D. of the Department of Pediatrics, is the lead
investiagor.
"We begin to see these patients, on average, about 10 years of age.
That’s because the build-up of materials takes several years before
it begins to demonstrate the symptoms," says Loew.
The symptoms include pain in the hands and feet, a dark-red skin rash,
and kidney and heart problems. Fabry's disease is often fatal by age 40.
Researchers want to determine the effectiveness of 'new' therapy for patients,
called fabrazyme.
"There is a great interest in the development of this enzyme because,
obviously, if we can reverse this process, and of course in newly diagnosed
individuals, prevent the whole process, then that will have great benefit
to these patients and their families."
About seven hundred people are affected by Fabry's disease in the United
States, inlcuding a handful of people in Iowa and the midwest. Loew believes
if this new therapy is successful, it will save thousands of lives worldwide.
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Children's Hospital
of Iowa
Thomas Loew, M.D.
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