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 TERMINÉ 

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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 TERMINÉ 

Orphan Drug for Acanthamoeba Keratitis (ODAK)

Date du début: 1 déc. 2012, Date de fin: 30 nov. 2017,

This project will undertake preclinical and clinical research of the Orphan Drug Polihexanide (PHMB). The main objective is to provide a safe and effective drug for the treatment of the rare ocular disease Acanthamoeba keratitis (AK) tested according to international regulatory standards. This debilitating infectious disease is caused by a free living protozoan which, in the absence of treatment ...
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