Recent results from trials initiated by the Neurofibromatosis Clinical Trials Consortium (NFCTC) have been extremely promising, demonstrating that Selumetinib (KOSELUGO, AstraZeneca, Cambridge, UK), a MEK inhibitor, can significantly reduce the size of NF1-associated plexiform neurofibromas.9 The gene discussed is MAP2K7; the disease is plexiform neurofibroma.