The discovery of PCSK9-based therapies began in 2003, with an astute clinical observation of a French family, which demonstrated features of familial hypercholesterolemia (FH), without mutations in the genes contemporaneously recognised to cause FH; the LDL receptor gene (LDLR, accounting for 95 % of FH defects), or apolipoprotein B gene, encoding the protein that binds to the LDLR (ApoB, accounting for 4 % of FH defects) [6, 7]. The gene discussed is PCSK9; the disease is familial hypercholesterolemia.