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Nitisinone
Nitisinone (also known as NTBC, an abbreviation of its full chemical name) is a drug used to slow the effects of hereditary tyrosinemia type 1. Since its first use for this indication in 1991, it has replaced liver transplantation as the first-line treatment for this rare condition. It is also being studied in the related condition alkaptonuria. It is marketed under the brand name Orfadin by the company, Swedish Orphan Biovitrum (Sobi) after having originally been brought to market by Swedish Orphan International. It was originally developed as candidate herbicide.
==Uses==
Nitisinone is used to treat hereditary tyrosinemia type 1, in combination with restriction of tyrosine in the diet.〔〔Sobi (Orfadin® (nitisinone) )〕
Since its first use for this indication in 1991, it has replaced liver transplantation as the first-line treatment for this rare condition. I It is marketed under the brand name Orfadin.
It has been demonstrated that treatment with nitisinone can reduce urinary levels of homogentisic acid in alkaptonuria patients by 95%. A series of clinical trials run by DevelopAKUre to determine whether nitisinone is effective at treating the ochronosis suffered by patients with alkaptonuria are ongoing. If the trials are successful, DevelopAKUre will try to get nitisinone licensed for use by alkaptonuria patients.
抄文引用元・出典: フリー百科事典『 ウィキペディア（Wikipedia）』
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