Pretreatment cognitive and neural differences between sapropterin dihydrochloride responders and non-responders with phenylketonuria

Sapropterin dihydrochloride (BH4) reduces phenylalanine (Phe) levels and improves white matter integrity in a subset of individuals with phenylketonuria (PKU) known as “responders.” Although prior research has identified biochemical and genotypic differences between BH4 responders and non-responders...

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Autori principali:	Zoë Hawks, Joshua Shimony, Jerrel Rutlin, Dorothy K. Grange, Shawn E. Christ, Desirée A. White
Natura:	Artigo
Lingua:	Inglês
Pubblicazione:	Elsevier 2017-09-01
Serie:	Molecular Genetics and Metabolism Reports
Soggetti:	Phenylketonuria Sapropterin dihydrochloride BH4 Intelligence IQ White matter Diffusion tensor imaging
Accesso online:	http://www.sciencedirect.com/science/article/pii/S2214426917300058
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