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Stem Cell Treatment May Have Cured Child With Rare Skin Disease

 
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University of Minnesota doctors believe they have hit a "home run" in using stem cell therapy in a 2-year-old boy's bone marrow by curing him of a rare disease that had been described as incurable.

The Minneapolis Star Tribune reports that doctors performed a bone marrow transplant on 2-year-old Nate Liao, who had been suffering from recessive dystrophic epidermolysis bullosa (RDEB), a genetic condition that literally causes skin to peel off at the slightest touch.

The stem cell procedure, previously done only in laboratory animals, was successful, the newspaper reports, so successful in fact, that University of Minnesota bone marrow specialist Dr. John Wagner said, "Maybe we can take one more disorder off the incurable list. It's not often that it feels like you hit a home run in medical research, but this one feels like it."

Nate's older brother Jake, who also has the disorder, was given a bone marrow transplant late last week, and 30 patients will be part of an upcoming clinical trial to continue the research, the Star Tribune reports.

RDEB falls into the category of orphan diseases -- very rare maladies. In this case, the skin disorder affects about 10 people per million.