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Trial MPS therapy gives sisters hope

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OREM — Speaking of miracles, Jeanene Underwood and her husband John believe they've had a fair share.

They have two daughters who've been diagnosed with MPS and who've already defied the odds by living past their life expectancy.

Jennifer, 14, and Jill, 12, both have Hurler-Sheie syndrome, a variation of the mucopolysaccharide disorder that slowly robs the body of its ability to shed discarded connective tissue.

The two sisters are involved in a clinical trial for enzyme replacement therapy.

Every week for six months they fly to New York City for an infusion.

"It's definitely a ray of hope," said their mother. "There hasn't really been anything before now other than a bone marrow transplant or surgery to relieve the stress on their joints."

If successful and approved by the Federal Food and Drug Administration for patient use, the enzyme replacement could be a small breakthrough for some MPS families with the same syndrome.

Ryan Dance, a young boy in California who's been given the drug over the past four years, is now walking and playing baseball. He was in a wheelchair before treatment.

The Underwoods and their daughters fly out every Tuesday for Wednesday appointments and return Thursday. Their expenses are paid by Genzyme and Bio-Marin, the drug companies sponsoring the trial.

The study is a blind study where half of the MPS children receive the enzyme and half do not, until the end of the trial.

After the study is complete, all of the children who've participated can get the drug from a site at the University of Utah.

Visible improvements? Underwood said the first changes are only detectable at the cellular level.

If the drug works, the liver will then shrink, hopefully signalling that the body is accepting the enzyme and returning to normal.

Underwood is cautiously optimistic for her daughters.

"I stumbled upon this after I saw a special on Ryan Dance where they talked about his progress. I checked the MPS Association Web site and was amazed. I didn't think there was anything (for these kids)!" she said.

"There's so much of this that's a puzzle. They just don't know much about it."


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