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...
Main Authors: | , , , , , |
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Format: | Article |
Language: | English |
Published: |
Elsevier
2017-09-01
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Series: | Molecular Genetics and Metabolism Reports |
Subjects: | |
Online Access: | http://www.sciencedirect.com/science/article/pii/S2214426917300058 |