Myelodysplastic syndromes (MDS) represent a clinically heterogeneous group of disorders with challenging unmet therapeutic needs. At the 20th Congress of the European Hematology Association (EHA), Valeria Santini, MD, of the University of Florence, Florence, Italy, discusses emerging agents for the treatment of patients with MDS, including transforming growth factor-beta inhibitors, thrombopoietic agents and targets of mutant IDH2.