Tag: Hereditary tyrosinemia type 1

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?

With annual costs for nitisinone treatment potentially running into the hundreds of thousands of dollars, managing the financial aspect of this rare disease medication is a significant concern for patients. This article explores the factors influencing how much does nitisinone cost and outlines the various pathways for financial relief.