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Ulodesine is a small molecule inhibitor of purine nucleoside phosphorylase (PNP), an enzyme involved in the purine metabolism pathway. By inhibiting PNP, ulodesine reduces the formation of uric acid by acting upstream of xanthine oxidase. This mechanism makes it a potential treatment for hyperuricemia and gout. Ulodesine has been studied as both monotherapy and in combination with allopurinol for chronic management of gout and has shown promising urate-lowering effects in Phase II clinical trials. It is orally active and suitable for once-daily dosing. The drug has also received orphan drug status for leukemia (second-line therapy or greater) in the USA and is being explored as an immunotherapy/antineoplastic agent due to its impact on T cells; lack of PNP activity can be associated with immunodeficiency[1][2][3][4][5][6][7].
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