The use of targeted therapeutics such as VEGF, mTOR, c-kit or MEK inhibitors in cNF is likely based on data suggesting that these targets are important in NF1-deficient tumours.115–118 Additionally, the overlap between pathways dysregulated by NF1 loss and pathways dysregulated in other cancer types has also inspired clinical trials that reposition existing cancer treatment options. This evidence concerns the gene NPHS1 and cancer.