Nitisinone is a medication that is primarily used to treat a rare genetic disorder known as hereditary tyrosinemia type 1 (HT-1). This condition prevents the body from breaking down the amino acid tyrosine, leading to a buildup of toxic byproducts that can cause serious liver and kidney damage if left untreated.

Nitisinone works by inhibiting an enzyme in the body that is responsible for the production of these harmful byproducts. By reducing the levels of these toxins, nitisinone can help to prevent the progression of liver and kidney damage in individuals with HT-1.

It is important to note that nitisinone is not a cure for HT-1, but rather a treatment that can help to manage the symptoms of the condition and improve quality of life. It is typically used in combination with a strict low-protein diet and other medications to help control the buildup of tyrosine in the body.

As with any medication, nitisinone can cause side effects in some individuals. Common side effects may include nausea, vomiting, diarrhea, and abdominal pain. More serious side effects such as liver toxicity or blood clotting disorders are rare but can occur.

If you or a loved one has been diagnosed with HT-1 and prescribed nitisinone, it is important to follow your healthcare provider’s instructions carefully and attend regular monitoring appointments to ensure the medication is working effectively and safely. Your healthcare provider can provide more information about the risks and benefits of nitisinone and help you make informed decisions about your treatment plan.