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104206-65-7
4-hydroxyphenylpyruvate dioxygenase inhibitor
DMF Active in US regulatory_status
NITYR, ORFADIN
Tyrosinemia
#035810
Nitisinone works by inhibiting the enzyme 4-hydroxyphenylpyruvate dioxygenase, which is involved in the catabolic pathway of the amino acid tyrosine. This prevents the accumulation of toxic metabolites in the blood and tissues.
Nitisinone is used to treat hereditary tyrosinemia type 1, a rare genetic metabolic disorder.
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