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Title

Long-term follow-up and outcome of phenylketonuria patients on sapropterin: a retrospective study

Authors
Keil, Stefanie
Anjema, Karen
van Spronsen, Francjan J
Lambruschini, Nilo
Burlina, Alberto
Bélanger-Quintana, Amaya
Couce, Maria L
Feillet, Francois
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Published in Pediatrics. 2013, vol. 131, no. 6, p. e1881-8
Abstract Sapropterin dihydrochloride, the synthetic form of 6R-tetrahydrobiopterin (BH4), is an approved drug for the treatment of patients with BH4-responsive phenylketonuria (PKU). The purpose of this study was to assess genotypes and data on the long-term effects of BH4/sapropterin on metabolic control and patient-related outcomes in 6 large European countries.
Keywords AdolescentAdultBiopterin/analogs & derivatives/therapeutic useChildChild, PreschoolDietEuropeFollow-Up StudiesGenotypeHumansInfantMiddle AgedMutationPhenotypePhenylalanine/bloodPhenylketonurias/blood/drug therapyQuestionnairesRetrospective StudiesTreatment OutcomeYoung Adult
Identifiers
PMID: 23690520
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Structures
Research group Gastroentérologie et transplantation (pédiatrie) (181)
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KEIL, Stefanie et al. Long-term follow-up and outcome of phenylketonuria patients on sapropterin: a retrospective study. In: Pediatrics, 2013, vol. 131, n° 6, p. e1881-8. https://archive-ouverte.unige.ch/unige:35270

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Deposited on : 2014-04-02

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