The Cincinnati Enquirer

A 4-year-old girl from Grayson, Ky., with a rare, often fatal disease will travel to Cincinnati today to receive a new therapy developed in part by Cincinnati Children's Hospital Medical Center.

Susie Blevins was diagnosed with MPS 1 when she was nine months old; the disorder affects about 3,000 children nationwide. Children with MPS 1 lack an enzyme that processes carbohydrates, which can lead to extensive organ damage. Children with the most severe form of MPS typically die before age 10.

Until recently, the only treatment option was a bone marrow transplant. But Susie will be among the first patients in the nation to get a new enzyme replacement therapy, called Aldurazyme, which was approved by the Food and Drug Administration on April 30 and was studied by researchers at Cincinnati Children's.

Although MPS-1 affects a small number of people, the research could lead to improved care for other enzyme-related disorders, which combined are five to seven times more common than childhood leukemia, said hospital spokesman Jim Feuer.