Start date: 2012-11-01, End date: 2018-04-30
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...
Record Number: 106157
Last updated on: 2016-06-27