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Nature Reviews Drug Discovery 7, 199–200 (1 March 2008) | doi:10.1038/nrd2540
Sapropterin
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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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