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What is Orfadin?
Nitisinone is used to treat a certain inherited disorder (hereditary tyrosinemia type 1, also known as HT-1). HT-1 is usually discovered in infants and needs lifelong treatment. This condition is caused by a shortage of a certain natural substance that is needed to break down a nutrient (tyrosine) found in food. This effect causes a build-up of too much tyrosine and related substances in the liver. Nitisinone works by helping to prevent the formation and build-up of several toxic substances that cause damage to the liver, kidneys, and nervous system. This drug must be used along with a diet low in protein, tyrosine, and phenylalanine.