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Comparing nitisinone 2 mg and 10 mg in the treatment of alkaptonuria-An approach using statistical modelling.

Lakshminarayan R RanganathAnna M MilanAndrew T HughesMilad KhedrBrendan P NormanMohammed AlsbouRichard ImrichMatthew GornallNicolas SireauJames A GallagherRichard Jackson
Published in: JIMD reports (2021)
Nitisinone 10 mg apparently slows alkaptonuria disease progression more than 2 mg in adults.Corneal keratopathy during nitisinone therapy was more common in men.Serum nitisinone concentrations increased significantly over time.Nitisinone may inhibit cytochrome P450 self catabolism.
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