Selumetinib has shown promising results in treating children with NF1 mutant plexiform neurofibromas (Dombi et al., 2016), while PD-0325901 has shown efficacy in treating NF1 mutant neurofibromas in mouse- and human-derived malignant peripheral nerve sheath xenografts (Jessen et al., 2013). This evidence concerns the gene NF1 and neurofibroma.