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University of Iowa News Release

Sept. 2, 2003

Children's Hospital of Iowa Research Benefits MPS I Patients

Children's Hospital of Iowa specialists are now treating patients with a new therapy they helped develop for patients diagnosed with a rare genetic disorder called mucopolysaccharidosis I (MPS I).

MPS I affects approximately 3,000 to 4,000 people in the world, including about 1,000 in the United States. It is a progressive, debilitating disorder caused by a deficiency of an enzyme called alpha L-iduronidase. People who lack this enzyme accumulate a carbohydrate called glycosaminoglycan in their tissues and organ systems. Most patients diagnosed with MPS I disorders die before reaching adulthood due to a wide range of complications caused by the disease, including progressive damage to the heart, lungs, liver and kidneys.

The Food and Drug Administration recently approved a medication called Aldurazyme, manufactured by BioMarin Pharmaceutical, Inc., and Genzyme General, as the first specific treatment for people affected by certain forms of MPS I. The therapy is used to treat patients with the Hurler and Hurler-Scheie forms of MPS I, and for Scheie patients with moderate to severe symptoms.

Thomas Loew, M.D., an assistant clinical professor with Children's Hospital of Iowa in University of Iowa Hospitals and Clinics, helped study Aldurazyme.

"We have observed remarkable improvements in the lives of patients after they receive this therapy," Loew said. "Before this enzyme replacement treatment became available, we could primarily only manage the wide array of symptoms in MPS I patients. Now, by addressing the underlying cause of the disorder, we have the opportunity to change the course of the disease for the better."

Studies showed that the therapy was effective in reducing the amount of glycosaminoglycan carbohydrates excreted in the urine, reducing liver size and increasing lung function. The research results suggest that the treatment is effective at a biochemical level. The medication replaces the missing enzyme in the body, allowing the patient to break down the carbohydrate and prevent the buildup that leads to tissue and organ damage.

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