[Comment] Does siRNA therapy against ANGPTL3 bring new hope in homozygous familial hypercholesterolaemia?

Homozygous familial hypercholesterolaemia is a rare and devastating autosomal co-dominant disorder affecting between one in 160 000 and 300 000 people in the general population.1 Homozygous familial hypercholesterolaemia is characterised by highly elevated LDL cholesterol concentrations from birth (usually >10 mmol/L). It can cause severe and diffuse atherosclerosis, xanthomas, and aortic or supraaortic valve stenosis during the first and second decades of life if left untreated.2,3 In most cases, the disease is caused by loss-of-function variants in the gene encoding the LDL receptor (LDLR) and, less commonly, by defects in the genes encoding apolipoprotein B (APOB) and PCSK9 (PCSK9).