Findings by a Saint Louis University research team led by the scientist who discovered Sly Syndrome 32 years ago point to a new direction for research into the rare genetic disorder that can cause bone deformities, vision and hearing loss, mental retardation and death in children.
The research is published in the Proceedings of the National Academy of Sciences Online Early Edition the week of Aug. 9.
“The importance of this research goes far beyond this rare disorder,” said William Sly, M.D., chairman of the department of biochemistry and molecular biology at Saint Louis University School of Medicine. “It could potentially provide access to the brain for enzyme therapy in other similar diseases, most of which are more common than Sly Syndrome.”
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