Tag: Nitisinone

First approved by the FDA in 2002, nitisinone has proven essential for managing rare inherited disorders. For those seeking to know **what is another name for nitisinone**, it is primarily known by its brand names, Orfadin and Nityr, and by the brand Harliku for a specific condition.

First approved by the FDA in 2002 for hereditary tyrosinemia type 1 (HT-1), nitisinone revolutionized the treatment of this life-threatening genetic disorder. But **which enzyme does nitisinone block** to achieve such a dramatic clinical outcome, and how does this mechanism prevent severe organ damage?

Nitisinone, originally developed as a herbicide, has dramatically changed the prognosis for patients with hereditary tyrosinemia type 1 since its first use in the 1990s. This article explores what is nitisinone used for, detailing its crucial role in managing specific rare metabolic disorders alongside necessary dietary adjustments.