Luspatercept is an FDA-approved recombinant fusion protein used to manage anemia in patients with β-thalassemia who require regular blood transfusions. This drug is also indicated for adults with very low-to-intermediate risk myelodysplastic syndromes (MDS) who are ESA-naïve and may need red blood cell transfusions. Luspatercept binds to transforming growth factor-β ligands, reducing SMAD2 and SMAD3 signaling to promote erythroid maturation.
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