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Clinical Development of Nitisinone for Alkaptonuria (DEVELOPAKURE)

Date du début: 1 nov. 2012, Date de fin: 30 avr. 2018,

DevelopAKUre is a proposal to fund the clinical development of an orphan designated drug, nitisinone, for the treatment of a rare Mendelian disease, Alkaptonuria (AKU). AKU is a genetic deficiency of homogentisic acid dioxygenase, causing high levels of homogentisic acid (HGA). Oxidation of HGA to pigment polymer, termed ochronosis, alters connective tissues. This leads to multisystemic damage dom ...
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