Nitisinone

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Nitisinone

Nitisinone is a medicinal drug used primarily in the treatment of hereditary tyrosinemia type 1 (HT-1). It is a synthetic compound of the hydroxyphenylpyruvate dioxygenase inhibitor class.

History

Nitisinone was initially developed in the 1980s as a herbicide by the Swedish company Kabi Pharmacia. However, its potential therapeutic use was discovered when it was found to be effective in treating hereditary tyrosinemia type 1 in experimental models.

Medical Uses

Nitisinone is used in the treatment of hereditary tyrosinemia type 1, a rare genetic disorder that can cause severe liver and kidney damage. It works by inhibiting the enzyme 4-hydroxyphenylpyruvate dioxygenase, thereby preventing the accumulation of toxic metabolites in the body.

Side Effects

Like all medications, Nitisinone can cause side effects. Common side effects include thrombocytopenia, leukopenia, granulocytopenia, conjunctivitis, corneal opacity, itching, exfoliative dermatitis, alopecia, and dry skin.

Pharmacology

Nitisinone inhibits the enzyme 4-hydroxyphenylpyruvate dioxygenase, which is involved in the catabolism of tyrosine. This inhibition prevents the formation of fumarylacetoacetate, a toxic metabolite that causes damage to the liver and kidneys in patients with hereditary tyrosinemia type 1.

See Also

• Hereditary tyrosinemia type 1
• Hydroxyphenylpyruvate dioxygenase
• Pharmacology