Nitisinone is an oral medication used to treat hereditary tyrosinemia type 1 (HT-1), a rare genetic disorder that affects the body's ability to break down the amino acid tyrosine. Without treatment, HT-1 can lead to severe complications, including liver disease, kidney damage, and neurological problems. Nitisinone works by blocking the enzyme that breaks down tyrosine, allowing the body to better process and utilize it.
Nitisinone is used to prevent the development of serious complications associated with HT-1. It can help reduce the amount of tyrosine in the blood and urine, as well as decrease the amount of certain toxic substances that can build up in the body. Nitisinone also helps the body to better absorb and use vitamins and minerals, and it may also help improve the body's ability to fight infections.
In addition to treating HT-1, nitisinone is also often prescribed for other disorders that can cause elevated tyrosine levels, such as Alagille syndrome and tyrosinemia type 2. Nitisinone has also been used to treat other conditions, such as non-alcoholic fatty liver disease, cystic fibrosis, and porphyria cutanea tarda.
It is important to note that nitisinone is not a cure for any of these conditions. It is only used to help manage the symptoms and improve quality of life. It is important to discuss all possible side effects and risks with your doctor before starting treatment with nitisinone.