PrMDK-Nitisinone by MendeliKABS

Treatment with Nitisinone was found to result in reduced risk for the development of hepatocellular carcinoma, compared to historical data on treatment with dietary restriction alone. Early initiation of treatment was associated with a further reduced risk for the development of hepatocellular carcinoma.


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Treatment with Nitisinone should be initiated and supervised by a physician experienced in the treatment of HT-1. Nitisinone is used for the treatment of hereditary tyrosinemia type 1 (HT-1), in addition to limiting the amount of tyrosine and phenylalanine in the diet. Nitisinone stops the build-up of toxic substances which cause the severe liver and kidney problems in patients with HT-1. By doing that, it also prevents the porphyric crises associated with HT-1.

Dietary intake of tyrosine and phenylalanine must be restricted during Nitisinone therapy. It is recommended that a dietician skilled in managing patients with inborn errors of metabolism be consulted to design a low-protein diet restricted in tyrosine and phenylalanine.

For more information about Nitisinone, talk to your healthcare professional or send us an email to request the Patient Medication Information or the Product Monograph.