No specific treatment currently availableMEK inhibitors (future perspective) In mouse models of Noonan syndrome, the administration of MEK inhibitors during 4–10 weeks of life could prevent the development of cardiomyopathy. Data from a clinical report showed that MEK inhibitors can induce the regression of cardiac hypertrophy within 4 months of treatment. mTOR inhibitors (future perspective) In mouse models with PTPN11 mutation Y279C, the administration of rapamycin, an inhibitor of mTOR–PI3K–AKT pathway, induced regression of HCM. The gene discussed is MTOR; the disease is Noonan syndrome.