Sapropterin

Barbara K. Burton*, Santwana Kar, Peter Kirkpatrick

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

4 Scopus citations

Abstract

Sapropterin dihydrochloride (Kuvan; BioMarin) is a synthetic form of tetrahydrobiopterin, the natural cofactor for the enzyme phenylalanine hydroxylase (PAH). In December 2007, it became the first drug to be approved by the US FDA for the treatment of phenylketonuria, a genetic disorder that is characterized by PAH deficiency.

Original languageEnglish (US)
Pages (from-to)199-200
Number of pages2
JournalNature Reviews Drug Discovery
Volume7
Issue number3
DOIs
StatePublished - Mar 2008

ASJC Scopus subject areas

  • Pharmacology
  • Drug Discovery

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