Indicated for: hereditary tyrosinemia type 1
Route of administration: oral
Substance: nitisinone (enzyme inhibitor)
ATC: A16AX04 (Alimentary tract and metabolism | Other alimentary tract and metabolism products | Various alimentary tract and metabolism products)
Nitisinone is a medication used for the treatment of hereditary tyrosinemia type 1, a rare metabolic disorder caused by an enzymatic deficiency. It works by inhibiting an enzyme involved in tyrosine metabolism, preventing the accumulation of toxic metabolites in the body.
The medication is taken orally, usually once or twice daily, with the dose adjusted based on the patient's weight and plasma tyrosine levels. Common side effects include elevated tyrosine levels, photosensitivity, and eye irritation.
Nitisinone is contraindicated in patients with hypersensitivity to the active substance. Additionally, it is important for patients to follow a strict low-tyrosine and low-phenylalanine diet to maximize the treatment's effectiveness.
This medication is an essential option for managing type 1 tyrosinemia, preventing severe complications and improving the quality of life for affected patients.