Two monoclonal antibodies, alirocumab and evolocumab, as well as the small interfering RNA Inclisiran, which inhibit PCSK9, have been approved for the treatment of hypercholesterolemia‐related cardiovascular diseases (CVD).[2] Beyond this established role in CVD, PCSK9 is being investigated for its involvement in a range of other pathological conditions, including cancer, infectious diseases, and neurodegenerative disorders. This evidence concerns the gene PCSK9 and familial hypercholesterolemia.