Sapropterin and stability of blood phenylalanine in PKU

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Variability in blood phenylalanine levels is inversely correlated with IQ and was recently shown to be a better predictor of IQ in early and continuously treated patients with PKU than mean blood phenylalanine levels. This study concludes that sapropterin results in increased stability of blood phenylalanine levels in BH4-responsive PKU.

Sapropterin therapy increases stability of blood phenylalanine levels in patients with BH4-responsive phenylketonuria (PKU). Burton et al. Mol Genet Metab. 2010 Oct-Nov;101(2-3):110-4. Epub 2010 Jun 27.

posted by Yannis Trakadis

 

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