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Arginine butyrate in combination with epoetin alfa is an experimental therapeutic regimen developed by Boston University for the treatment of beta-thalassemia intermedia. Arginine butyrate is a small molecule short-chain fatty acid derivative that acts as a fetal globin gene inducer, working primarily by inhibiting histone deacetylation to stimulate gamma-globin chain production. Epoetin alfa (also known as recombinant human erythropoietin or EPO) is a biologic hematopoietic growth factor that prolongs erythroid cell survival and stimulates red blood cell production. The combination therapy was evaluated in Phase II clinical trials to determine whether the two agents could produce additive hematologic responses by increasing fetal hemoglobin levels and enhancing erythropoiesis in patients with thalassemia intermedia, particularly those with relatively low baseline endogenous EPO levels.
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