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Nitisinone
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Nitisinone
Nitisinone, sold under the brand name Orfadin among others, is a medication used for the treatment of hereditary tyrosinemia type 1; or for the reduction of urine homogentisic acid in adults with alkaptonuria. Nitisinone is a hydroxyphenyl-pyruvate dioxygenase inhibitor.
It is available as a generic medication.
Nitisinone (Nityr, Orfadin) is indicated for the treatment of hereditary tyrosinemia type 1 in combination with dietary restriction of tyrosine and phenylalanine. Nitisinone (Harliku) is also indicated for the reduction of urine homogentisic acid in adults with alkaptonuria.
Nitisinone is used to treat hereditary tyrosinemia type 1 (HT-1) and alkaptonuria (AKU) in patients from all ages, in combination with dietary restriction of tyrosine and phenylalanine.
Since its first use for this indication in 1991, it has replaced liver transplantation as the first-line treatment for this condition.
It has been shown that nitisinone is toxic to kissing bugs, tsetse, ticks and mosquitoes.. The substance may be in the host's bloodstream, or spread on surfaces, as it is absorbed through the mosquito's skin.
The most common adverse reactions (>1%) for nitisinone are elevated tyrosine levels, thrombocytopenia, leukopenia, conjunctivitis, corneal opacity, keratitis, photophobia, eye pain, blepharitis, cataracts, granulocytopenia, epistaxis, pruritus, exfoliative dermatitis, dry skin, maculopapular rash and alopecia. Nitisinone has several negative side effects; these include but are not limited to: bloated abdomen, dark urine, abdominal pain, feeling of tiredness or weakness, headache, light-colored stools, loss of appetite, weight loss, vomiting, and yellow-colored eyes or skin.
The mechanism of action of nitisinone involves inhibition of 4-Hydroxyphenylpyruvate dioxygenase (HPPD). This is a treatment for patients with Tyrosinemia type 1 as it prevents the formation of 4-Maleylacetoacetic acid and fumarylacetoacetic acid, which have the potential to be converted to succinyl acetone, a toxin that damages the liver and kidneys.
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Nitisinone
Nitisinone, sold under the brand name Orfadin among others, is a medication used for the treatment of hereditary tyrosinemia type 1; or for the reduction of urine homogentisic acid in adults with alkaptonuria. Nitisinone is a hydroxyphenyl-pyruvate dioxygenase inhibitor.
It is available as a generic medication.
Nitisinone (Nityr, Orfadin) is indicated for the treatment of hereditary tyrosinemia type 1 in combination with dietary restriction of tyrosine and phenylalanine. Nitisinone (Harliku) is also indicated for the reduction of urine homogentisic acid in adults with alkaptonuria.
Nitisinone is used to treat hereditary tyrosinemia type 1 (HT-1) and alkaptonuria (AKU) in patients from all ages, in combination with dietary restriction of tyrosine and phenylalanine.
Since its first use for this indication in 1991, it has replaced liver transplantation as the first-line treatment for this condition.
It has been shown that nitisinone is toxic to kissing bugs, tsetse, ticks and mosquitoes.. The substance may be in the host's bloodstream, or spread on surfaces, as it is absorbed through the mosquito's skin.
The most common adverse reactions (>1%) for nitisinone are elevated tyrosine levels, thrombocytopenia, leukopenia, conjunctivitis, corneal opacity, keratitis, photophobia, eye pain, blepharitis, cataracts, granulocytopenia, epistaxis, pruritus, exfoliative dermatitis, dry skin, maculopapular rash and alopecia. Nitisinone has several negative side effects; these include but are not limited to: bloated abdomen, dark urine, abdominal pain, feeling of tiredness or weakness, headache, light-colored stools, loss of appetite, weight loss, vomiting, and yellow-colored eyes or skin.
The mechanism of action of nitisinone involves inhibition of 4-Hydroxyphenylpyruvate dioxygenase (HPPD). This is a treatment for patients with Tyrosinemia type 1 as it prevents the formation of 4-Maleylacetoacetic acid and fumarylacetoacetic acid, which have the potential to be converted to succinyl acetone, a toxin that damages the liver and kidneys.
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