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Also known as hereditary tyrosinemia type 1 (HT-1), tyrosinemia is a rare, lifelong disorder where the body cannot break down a substance called tyrosine. Tyrosine is one of 20 amino acids that are the building blocks of protein found in food.
Tyrosinemia, if left untreated, can lead to growth and developmental dysfunction throughout life
If untreated, complications of tyrosinemia can cause death before the age of 10 years
Worldwide, around 1 in every 100,000 babies will have tyrosinemia
Males and females have an equal chance of developing tyrosinemia
Tyrosinemia is an inherited disorder. It is passed on through parents when they both have non-working copies of the tyrosinemia gene
Orfadin is a synthetic reversible inhibitor of 4-hydroxyphenylpyruvate dioxygenase indicated for use as an adjunct to dietary restriction of tyrosine and phenylalanine in the treatment of hereditary tyrosinemia type 1 (HT-1).