The new therapies targeting apoB metabolism for high-risk patients with familial hypercholesterolemia, such as mipomersen to inhibit expression of hepatic apoB and lomitapide to block hepatic MTP activity, have demonstrated excellent efficacy for reducing atherogenic lipoproteins but have also significantly raised the risk of hepatic transaminase (10 %) and hepatic steatosis (18 %) in users. The gene discussed is MTTP; the disease is fatty liver disease.