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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...
Autores principales: | , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Elsevier
2017
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5323508/ https://www.ncbi.nlm.nih.gov/pubmed/28271047 http://dx.doi.org/10.1016/j.ymgmr.2017.01.013 |