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Article

Sapropterin dihydrochloride for the treatment of hyperphenylalaninemias.

Expert opinion on drug metabolism & toxicology - 1 Sept 2013

Blau Nenad

Abstract excerpt

INTRODUCTION: Phenylketonuria (PKU) is caused by mutation of the enzyme, phenylalanine (Phe) hydroxylase (PAH). The hyperphenylalaninemia characteristic of PKU causes devastating neurological damage if not identified and treated at birth with a Phe-restricted diet. Sapropterin dihydrochloride, a pharmaceutical formulation of the natural cofactor for PAH (6R-tetrahydrobiopterin; BH4), is now available for the...

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Identifiers

PMID: 23705856
DOI: 10.1517/17425255.2013.804064

Genes

PAH

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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