Patients with a rare genetic disorder have new hope, thanks to a specially designed therapy. A pediatrician from University of Iowa University of Iowa Children's Hospital helped study the medication:
For people with the rare condition known as MPS I, there have been few treatment options to overcome this fatal condition. Today, there's new hope for these patients.
MPS-1 is a rare, inherited disorder that claims the lives of a majority of patients before they reach adulthood. Patients like Ryan Dant lack a particular enzyme, which causes a carbohydrate to accumulate in their tissues and organ systems.
"Ryan was diagnosed with MPS-1 at the age of 3. His liver and spleen were found to be enlarged, and as time went on, it would go to be 2 times normal size. He was unable to breathe normally," says Ryan's father, Mark Dant.
Today there's a new treatment for patients like Ryan. In fact, his condition improved when he began taking aldurazyme, an enzyme replacement therapy recently approved by the Food and Drug Administration. It's the first therapy to specifically treat the underlying cause of MPS-1. Tom Loew, M.D., a pediatrician with University of Iowa University of Iowa Children's Hospital, helped study aldurazyme.
"You have to have individuals who are familiar with the potential toxicities and also familiar with the disease and how the drug is administered. We have all of that capability right here at University of Iowa Children's Hospital," says Loew.
"The opportunity of having enzyme replacement therapy in his life has changed our life tremendously. Because now we all have a future together," says Mark Dant.
Because it's a progressive disorder, specialists believe early treatment may prevent the damage caused by MPS-1. So far, aldurazyme is making a big difference in Ryan's life.
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