Nitirisinone is a chemical compound originally developed as an herbicide, but now being investigated as a treatment for certain forms of type 1 tyrosinaemia. The mechanism of action of nitrisinone involves inhibition of the enzyme 4-hydroxyphenylpyruvic acid oxidase, thus preventing the formation of maleylacetoacetic acid and fumarylacetoacetic acid, which have the potential to be converted to succinyl acetone, a toxin that damages the liver and kidneys.
Additional recommended knowledge
- Koelink, C. J. L.; van Hasselt, P.; van der Ploeg, A.; van den Heuvel-Eibrink, M. M.; Wijburg, F. A.; Bijleveld, C. M. A.; van Spronsen, F. J. Tyrosinemia type I treated by NTBC: How does AFP predict liver cancer? Molecular Genetics and Metabolism (2006), 89(4), 310-315.
- McKiernan, Patrick J. Nitisinone in the treatment of hereditary tyrosinaemia type 1. Drugs (2006), 66(6), 743-750.
- Kavana, Michael; Moran, Graham R. Interaction of (4-hydroxyphenyl)pyruvate dioxygenase with the specific inhibitor 2-[2-nitro-4-(trifluoromethyl)benzoyl]-1,3-cyclohexanedione. Biochemistry (2003), 42(34), 10238-10245.