Discover Community Create You

Loading publication...

Noodle always counts identifier-free feature events for service operation. With your permission, Google Analytics can also estimate audience and engagement. Search text, publication identifiers, cookies, IP addresses and browser fingerprints are never included in Noodle's first-party event counters.

Privacy details

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 under the age of 4 years and demonstrate the safety and efficacy of this treatment for patients in this age group....

Read the complete abstract on PubMed

Topics

Share this publication in a Topic to start or enrich a Post.

Share to Age Factors
Share to Biopterins

Methodology Data sources Editorial policy Privacy Delete account & data About Contact Helena Bioinformatics

Share to France

Share to Infant, Newborn

Share to Phenylalanine

Share to Phenylalanine Hydroxylase

Share to Phenylketonurias

Share to Treatment Outcome

Source

Identifiers

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

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence. Graph snapshot: 2026-09-29.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Efficacy and safety of BH4 before the age of 4 years in patients with mild phenylketonuria.PMID 22388642Read ↗

Sapropterin dihydrochloride for the treatment of hyperphenylalaninemias.Semantic proximity Read ↗

Effect of BH(4) supplementation on phenylalanine tolerance.Semantic proximity Read ↗

Clinical and nutritional evaluation of phenylketonuric patients on tetrahydrobiopterin monotherapy.Semantic proximity Read ↗

Neonatal phenylalanine wash-out in phenylketonuria.Semantic proximity Read ↗

Nutritional and Therapeutic Strategies in Paediatric Phenylketonuria: A Narrative Literature ReviewSemantic proximity Read ↗

Positive effect of a simplified diet on blood phenylalanine control in different phenylketonuria variants, characterized by newborn BH4 loading test and PAH analysis.Semantic proximity Read ↗

An Overview of Traditional and Novel Therapeutic Options for the Management of Phenylketonuria.Semantic proximity Read ↗

Optimizing the use of sapropterin (BH(4)) in the management of phenylketonuria.Semantic proximity Read ↗

Long-term follow-up of patients with phenylketonuria treated with tetrahydrobiopterin: a seven years experience.Semantic proximity Read ↗

Evaluation of neonatal BH4 loading test in neonates screened for hyperphenylalaninemia.Semantic proximity Read ↗

Long-term treatment with tetrahydrobiopterin increases phenylalanine tolerance in children with severe phenotype of phenylketonuria.Semantic proximity Read ↗

Does the 48-hour BH4 loading test miss responsive PKU patients?Semantic proximity Read ↗

⌁Select a neighboring publication to make it the new centre.