Patients affected by a rare and potentially fatal disease are now benefiting from a new treatment. Specialists at Children's Hospital of Iowa helped research this new medical breakthrough:

Fabry disease affects about 5,000 people worldwide, leading to kidney failure, heart disease, and ultimately death. A University of Iowa Health Care pediatrician is a leading researcher in the search for a cure.

Tom Loew, M.D., a pediatrician at University of Iowa Children's Hospital, helped conduct research that led to a new treatment for Fabry disease. The drug, fabrazyme, was recently approved by the food and drug administration. The therapy replaces an enzyme that Fabry patients are missing. Experts believe this therapy may eliminate some of the complications of the disease.

"There's still a lot of work to go," says Loew. "This is the type of thing that it took many, many years for these patients to develop their symptoms; it took many, many years for the damage to occur in their bodies, so it may take many years to reverse that process."

Fabry disease is usually diagnosed at an early age, but during adulthood it can lead to kidney failure, stroke, heart disease, disabling pain and sometimes death .

"We're going to get a very, very broad picture of exactly how effective this medication is going to be. We're very hopeful it will ultimately be very effective for these patients and prolong their lives and begin to reverse the damage," says Loew.

Researchers believe the next step involves treating children immediately following diagnosis of the disease, hopefully stopping the effects of Fabry disease before they start.

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Children's Hospital of Iowa

Tom Loew, M.D.