MEK inhibitors are being investigated in the clinic for treating symptomatic and inoperable plexiform neurofibromas in NF1 patients, with PD325901 treatment demonstrating neurofibroma shrinkage in Phase 2 trials in adolescents and adults [56], and a Phase 2b trial currently following up this result in children and adults (NCT03962543). The gene discussed is MAP2K7; the disease is neurofibroma.