The recent FDA approval of the mitogen-activated protein kinase kinase 1/2 (MEK1/2) inhibitor selumetinib for inoperable plexiform neurofibromas is one notable exception, although there are still improvements to be made with regard to adverse events and variability in response, and MEK inhibition may not be sufficient for other NF1-associated tumors like MPNST or juvenile myelomonocytic leukemia (JMML) [12,13,14]. The gene discussed is MAP2K1; the disease is juvenile myelomonocytic leukemia.