Nitisinone is an effective treatment for Hereditary Tyrosinemia Type 1 (HT-1), a rare metabolic disorder that affects the breakdown of the amino acid tyrosine. HT-1 can lead to liver, kidney, and nervous system complications. Nitisinone works by inhibiting the enzyme 4-hydroxyphenylpyruvate dioxygenase (HPPD), which prevents the accumulation of toxic byproducts in the body, particularly in the liver and kidneys.
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