The FDA-approved medicine nitisinone is used to treat HT-1 (hereditary tyrosinemia). It is a reversible synthetic inhibitor of 4-hydroxyphenylpyruvate dioxygenase, an enzyme that generates toxic chemicals related to HT-1. Patients must follow a rigorous diet that limits their intake of tyrosine and phenylalanine in addition to taking Nitisinone. Any unexpected eye issues, rashes, jaundice, or significant bleeding should be reported immediately. Nitisinone dosage can be changed based on how each patient responds to it, with a daily dose cap of 2 mg/kg. To minimize negative outcomes, plasma tyrosine levels must be carefully monitored.
More information about administering Nitisinone 2 mg to patients is available at

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