nitisinone


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nitisinone

an orphan drug.
indication This drug is used to treat hereditary tyrosinemia type 1.
contraindications Tyrosine or phenylalanine intake and known hypersensitivity to this drug prohibit its use.
References in periodicals archive ?
In a pioneering trial at Royal Liverpool Hospital, patients with alkaptonuria (AKU) - known as black bone disease - are being given a drug called nitisinone.
23] having noted that nitisinone (used in the treatment of hereditary tyrosinemia type 1) caused an increase in serum tyrosine levels, treated OCA1B mice with the drug and noted an improvement in pigmentation of the mice.
Nitisinone improves eye and skin pigmentation defects in a mouse model of oculocutaneous albinism.
Unlicensed drug nitisinone has had success in relieving symptoms during trials.
AKU Society head Dr Nicolas Sireau, who has two children with the disease, believes nitisinone is the answer.
And now, after his long and painstaking research, he told the Mirror: "We've found an amazing treatment, a drug called nitisinone.
A drug called nitisinone has also been mentioned as a potential treatment for AKU; however, the long-term clinical efficacy and safety of this drug have also not been demonstrated.
He eventually found an advert for a trial in Washington to test the impact of an existing drug, nitisinone.
Scientists at the National Eye Institute made this discovery as they investigated nitisinone, a drug that is currently approved for use in treating a blood condition, but is also known to increase hair and eye pigmentation.
Nashville, TN has received approval from the FDA to market nitisinone capsules under the name of Orfadin to treat hereditary tyrosinemia type 1 (HT-1), a rare pediatric disease causing progressive liver failure and liver cancer in young children.
Until now there has been no treatment for AKU, it is hoped the drug Nitisinone could alleviate the joint and back pain associated with the disease and, if given early enough, could allow sufferers to lead a normal, pain-free life.