The Food and Drug Administration (FDA) also approved APR-246 combined with azacitidine for the treatment of Li-Fraumeni syndrome complicated with MDS in January 2020. A recent study showed that APR-246 and azacitidine play a synergistic role in TP53-mutated MDS/AML, which can restore the transcriptional activation function of mutant TP53 and induce apoptosis of human tumor cells (14), and preclinical trials are currently underway. The gene discussed is TP53; the disease is myelodysplastic syndrome.