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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 ...

"Chronic inflammatory diseases of joints are major causes of disability in the ageing population. Osteoarthritis (OA) is one of the most common types of arthritis and a major cause of pain and disability in older individuals. OA is expected to place a heavy burden on European healthcare systems, as European citizens grow older. Cartilage damage in OA is detected radiographically by decreases in jo ...