Nitisinone is used to treat a rare genetic condition called hereditary tyrosinemia type 1 (HT-1). HT-1 is a metabolic disorder that occurs when the body does not produce enough of an enzyme that breaks down proteins from certain foods. This condition occurs most often in young babies. The symptoms of HT-1 include unusual bleeding and signs of liver failure, such as dark urine, jaundice (yellowing of the skin or eyes), sudden stomach pain, nausea, vomiting, diarrhea, and fatigue.
Nitisinone works by preventing the body from breaking down an amino acid called tyrosine and by keeping other toxic substances from building up and causing harm to your liver or kidneys.
- Orfadin Information for Consumers
- Orfadin Information for Healthcare Professionals (includes dosage details)
- Side Effects of Orfadin (detailed)
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