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Article

Efficacy and safety of BH4 before the age of 4 years in patients with mild phenylketonuria.

Journal of inherited metabolic disease - 1 Nov 2012

Leuret Oriane, Barth Magalie, Kuster Alice, Eyer Didier, de Parscau Loïc, Odent Sylvie, Gilbert-Dussardier Brigitte, Feillet François, Labarthe François

Abstract excerpt

BACKGROUND: Sapropterin dihydrochloride, an EMEA-approved synthetic formulation of BH4, has been available in Europe since 2009 for PKU patients older than 4 years, but its use with younger children is allowed in France based on an expert recommendation. We report the cases of 15 patients treated...

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Source

Identifiers

PMID: 22388642
DOI: 10.1007/s10545-012-9464-3

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 4 Sept 2026. Prepared record publication-22388642-2026-09-04.2. Manifest publication-graph-cohort-2026-09-04.2.

Source receipt: PubMed via Helena Literature Corpus, retrieved 4 Sept 2026.

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