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University of Iowa News Release

Aug. 29, 2003

Children's Hospital Of Iowa Research Benefits Fabry Patients

Photo: Lowe

Patients affected by a rare and potentially fatal inherited metabolic disorder called Fabry disease are now benefiting from a new treatment that researchers at Children's Hospital of Iowa helped create.

Fabry disease affects approximately 5,000 people worldwide. The average lifespan of people diagnosed with the disorder is 50 years. The disease is caused by a deficiency of an enzyme called alpha-galactosidase A. Patients affected by the condition can develop kidney failure, strokes, heart disease and disabling pain.

The Food and Drug Administration (FDA) recently approved a medication called Fabrazyme, produced by Genzyme General, for the treatment of Fabry disease. The therapy replaces the missing enzyme in order to help correct the disorder. The treatment took 30 years of research to develop.

Thomas Loew, M.D., associate professor (clinical) at Children's Hospital of Iowa in University of Iowa Hospitals and Clinics, helped conduct the research that led to the FDA's approval of Fabrazyme.

"This is truly good news for patients affected by Fabry disease. The FDA approval of this therapy marks a true milestone in the effort to ease the often devastating impact of this disorder upon patients and their families," Loew said.

Additional research is currently under way to determine if the treatment prevents or slows the development of the serious complications of Fabry disease.

Symptoms of Fabry disease usually begin during childhood. The disorder occurs in an estimated one out of every 40,000 males. A blood test can determine if someone is a carrier of Fabry disease.

Children's Hospital of Iowa at UI Hospitals and Clinics in Iowa City is the state's longest-serving children's hospital. More than 100,000 children receive care at the Children's Hospital of Iowa and its statewide network of outreach clinics each year.

University of Iowa Health Care describes the partnership between the UI Roy J. and Lucille A. Carver College of Medicine and UI Hospitals and Clinics and the patient care, medical education and research programs and services they provide. Visit UI Health Care online at www.uihealthcare.com.

STORY SOURCE: Joint Office for Planning, Marketing and Communications, University of Iowa Health Care, 200 Hawkins Drive, Room 8798 JPP, Iowa City, Iowa 52242-1009.

MEDIA CONTACT: Tom Moore, 319-356-3945, thomas-moore@uiowa.edu