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

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...

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Библиографические подробности
Главный автор: Harding, Cary O
Формат: Artigo
Язык:Inglês
Опубликовано: Dove Medical Press 2010
Предметы:
Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC2921259/
https://ncbi.nlm.nih.gov/pubmed/20714359
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