An open-label drug trial has been started with the mTOR inhibitor sirolimus (alternative name for rapamycin) to treat PHTS patients, and this drug has shown some efficacy in inhibiting the growth of lipomas, which are a type of hamartoma (Leipert et al., 2016; Schmid et al., 2014; Wang et al., 2007). The gene discussed is MTOR; the disease is hamartoma.