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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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Author notes

  1. Santwana Kar is at IMS Health, 7 Harewood Avenue, London NW1 6JB, UK. skar@uk.imshealth.com

  2. Peter Kirkpatrick is at Nature Reviews Drug Discovery. p.kirkpatrick@nature.com

Authors and Affiliations

  1. Barbara K. Burton is at the Department of Pediatrics, Children's Memorial Hospital, Northwestern University, Feinberg School of Medicine, 2300 Children's Plaza, Box 59, Chicago, Illinois 60614, USA. BBurton@childrensmemorial.org,

    Barbara K. Burton, Santwana Kar & Peter Kirkpatrick

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  1. Barbara K. Burton
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  2. Santwana Kar
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  3. Peter Kirkpatrick
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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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