Background: Familial hypercholesterolemia (FH) is associated with a 10-20 fold increase in cardiovascular (CV) events. Mipomersen has been shown to significantly lower levels of atherogenic lipoproteins in plasma. Previous safety analysis of all patients in phase 3 trials found no meaningful imbalance in CV events between placebo and mipomersen arms. Hypothesis: Treatment with mipomersen for at least 1 year will reduce CV events in FH patients taking maximally tolerated lipid-lowering therapy. Methods: Rates of major adverse CV events (MACE) during 2 years prior to mipomersen treatment were compared to MACE after treatment in 104 FH patients who participated in one of three phase 3 blinded randomized placebo-controlled 6-month trials and an open-label extension study (NCT00607373, NCT00706849, NCT00794664, NCT00694109). One third of patients (n=34) received placebo for the initial 6 months followed by mipomersen for at least 1 year. Two thirds (n=70) received blinded mipomersen for 6 months followed by at...