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Introduction of sapropterin dihydrochloride as standard of care in patients with phenylketonuria

Sapropterin dihydrochloride, a synthetic, stable form of the tetrahydrobiopterin cofactor of phenylalanine hydroxylase, has been shown to reduce plasma phenylalanine (Phe) levels in a significant portion of patients with phenylketonuria (PKU). When we undertook introducing this medication to our PKU...

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Bibliografiske detaljer
Main Authors: Vernon, HJ, Koerner, C, Johnson, M, Bergner, A, Hamosh, A
Format: Artigo
Sprog:Inglês
Udgivet: 2010
Fag:
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC3135651/
https://ncbi.nlm.nih.gov/pubmed/20418136
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2010.03.022
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