New era in treatment for phenylketonuria: Pharmacologic therapy with sapropterin dihydrochloride

Cary O HardingDepartments of Molecular and Medical Genetics and Pediatrics, Oregon Health &amp; Science University, Portland, Oregon, USAAbstract: Oral administration of sapropterin hydrochloride, recently approved for use by the US Food and Drug Administration and the European Commission, i...

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Autor principal:	Cary O Harding
Formato:	article
Lenguaje:	EN
Publicado:	Dove Medical Press 2010
Materias:	Medicine (General) R5-920
Acceso en línea:	https://doaj.org/article/8ad2204159d240fd8108f9cd8765077a
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spelling	oai:doaj.org-article:8ad2204159d240fd8108f9cd8765077a2021-12-02T03:49:15ZNew era in treatment for phenylketonuria: Pharmacologic therapy with sapropterin dihydrochloride1177-54751177-5491https://doaj.org/article/8ad2204159d240fd8108f9cd8765077a2010-08-01T00:00:00Zhttp://www.dovepress.com/new-era-in-treatment-for-phenylketonuria-pharmacologic-therapy-with-sa-a4989https://doaj.org/toc/1177-5475https://doaj.org/toc/1177-5491Cary O HardingDepartments of Molecular and Medical Genetics and Pediatrics, Oregon Health &amp; Science University, Portland, Oregon, USAAbstract: Oral administration of sapropterin hydrochloride, recently approved for use by the US Food and Drug Administration and the European Commission, is a novel approach for the treatment of phenylketonuria (PKU), one of the most common inborn errors of metabolism. PKU is caused by an inherited deficiency of the enzyme phenylalanine hydroxylase (PAH), and the pathophysiology of the disorder is related to chronic accumulation of the free amino acid phenylalanine in tissues. Contemporary therapy is based upon restriction of dietary protein intake, which leads to reduction of blood phenylalanine levels. This therapy is difficult to maintain throughout life, and dietary noncompliance is commonplace. Sapropterin dihydrochloride is a synthetic version of tetrahydrobiopterin, the naturally occurring pterin cofactor that is required for PAH-mediated phenylalanine hydroxylation. In a subset of individuals with PAH deficiency, sapropterin administration leads to reduction in blood phenylalanine levels independent of dietary protein. For these individuals, sapropterin is an effective novel therapy for PKU.Keywords: sapropterin dihydrochloride, phenylketonuria, phenylalanine, tetrahydrobiopterin Cary O HardingDove Medical PressarticleMedicine (General)R5-920ENBiologics: Targets & Therapy, Vol 2010, Iss default, Pp 231-236 (2010)
institution	DOAJ
collection	DOAJ
language	EN
topic	Medicine (General) R5-920
spellingShingle	Medicine (General) R5-920 Cary O Harding New era in treatment for phenylketonuria: Pharmacologic therapy with sapropterin dihydrochloride
description	Cary O HardingDepartments of Molecular and Medical Genetics and Pediatrics, Oregon Health &amp; Science University, Portland, Oregon, USAAbstract: Oral administration of sapropterin hydrochloride, recently approved for use by the US Food and Drug Administration and the European Commission, is a novel approach for the treatment of phenylketonuria (PKU), one of the most common inborn errors of metabolism. PKU is caused by an inherited deficiency of the enzyme phenylalanine hydroxylase (PAH), and the pathophysiology of the disorder is related to chronic accumulation of the free amino acid phenylalanine in tissues. Contemporary therapy is based upon restriction of dietary protein intake, which leads to reduction of blood phenylalanine levels. This therapy is difficult to maintain throughout life, and dietary noncompliance is commonplace. Sapropterin dihydrochloride is a synthetic version of tetrahydrobiopterin, the naturally occurring pterin cofactor that is required for PAH-mediated phenylalanine hydroxylation. In a subset of individuals with PAH deficiency, sapropterin administration leads to reduction in blood phenylalanine levels independent of dietary protein. For these individuals, sapropterin is an effective novel therapy for PKU.Keywords: sapropterin dihydrochloride, phenylketonuria, phenylalanine, tetrahydrobiopterin
format	article
author	Cary O Harding
author_facet	Cary O Harding
author_sort	Cary O Harding
title	New era in treatment for phenylketonuria: Pharmacologic therapy with sapropterin dihydrochloride
title_short	New era in treatment for phenylketonuria: Pharmacologic therapy with sapropterin dihydrochloride
title_full	New era in treatment for phenylketonuria: Pharmacologic therapy with sapropterin dihydrochloride
title_fullStr	New era in treatment for phenylketonuria: Pharmacologic therapy with sapropterin dihydrochloride
title_full_unstemmed	New era in treatment for phenylketonuria: Pharmacologic therapy with sapropterin dihydrochloride
title_sort	new era in treatment for phenylketonuria: pharmacologic therapy with sapropterin dihydrochloride
publisher	Dove Medical Press
publishDate	2010
url	https://doaj.org/article/8ad2204159d240fd8108f9cd8765077a
work_keys_str_mv	AT caryoharding neweraintreatmentforphenylketonuriapharmacologictherapywithsapropterindihydrochloride
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New era in treatment for phenylketonuria: Pharmacologic therapy with sapropterin dihydrochloride

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