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.
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References
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Competing interests
Barbara K. Burton has received research funding from BioMarin for clinical trials in PKU involving sapropterin.
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Burton, B., Kar, S. & Kirkpatrick, P. Sapropterin. Nat Rev Drug Discov 7, 199–200 (2008). https://doi.org/10.1038/nrd2540
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DOI: https://doi.org/10.1038/nrd2540
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