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Nationwide Children's Hospital involved in expanded access program for treatment of PKU

Nationwide Children's Hospital announced today its involvement in an expanded access program for sapropterin dihydrochloride, or sapropterin, an investigational treatment for phenylketonuria, a rare genetic metabolic disorder that, if left unmanaged in infants and children, can result in severe mental retardation. Currently, there are eight patients enrolled in the expanded access program at Nationwide Children's.

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Keywords: nationwide, children, hospital, involved, expanded, access, program, treatment, pku

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