For example, there are a number of trials including rapamycin/everolimus, targeting the mTOR pathway, for the treatment of tuberous sclerosis complex and related WS (43); retigabine for the treatment of KCNQ2-related WS (44); and topiramate for the treatment of CACNA1E-related WS (31). The gene discussed is MTOR; the disease is tuberous sclerosis.