Nitisinone (MDK) by MendeliKABS
Treatment with Nitisinone (MDK) should be initiated and supervised by a physician experienced in the treatment of HT-1. Nitisinone (MDK) 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 (MDK) 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.
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.
Dietary intake of tyrosine and phenylalanine must be restricted during Nitisinone (MDK) 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.
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