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

Sapropterin dihydrochloride (BH(4)) 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 BH(4) responders and non-respon...

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Detalhes bibliográficos
Publicado no:Mol Genet Metab Rep
Main Authors: Hawks, Zoë, Shimony, Joshua, Rutlin, Jerrel, Grange, Dorothy K., Christ, Shawn E., White, Desirée A.
Formato: Artigo
Idioma:Inglês
Publicado em: Elsevier 2017
Assuntos:
Research Paper
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5323508/
https://ncbi.nlm.nih.gov/pubmed/28271047
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgmr.2017.01.013
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