Nitisinone arrests ochronosis and decreases rate of progression of Alkaptonuria: Evaluation of the effect of nitisinone in the United Kingdom National Alkaptonuria Centre.
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DOI: 10.1016/j.ymgme.2018.07.011
Abstract: QUESTION Does Nitisinone prevent the clinical progression of the Alkaptonuria? FINDINGS In this observational study on 39 patients, 2 mg of daily nitisinone inhibited ochronosis and significantly slowed the progression of AKU over a three-year period.… read more here.
Keywords: aku; nitisinone; group; progression alkaptonuria ... See more keywords