New Frontiers in the Treatment of Homozygous Familial Hypercholesterolemia
Homozygous familial hypercholesterolemia (HoFH) is a rare genetic disorder. The most common cause is a mutation in both alleles of the gene encoding for the low-density lipoprotein (LDL) receptor, although other causative mutations have been identified. Complications of atherosclerotic cardiovascular disease are common in these patients; therefore, reducing the elevated LDL-cholesterol burden is critical in their management. Conventionally, this is achieved by patients initiating lipid-lowering therapy, but this can present challenges in clinical practice. Fortunately, novel therapeutic strategies have enabled promising innovations in HoFH treatment. This review highlights recent and ongoing studies examining new therapeutic options for patients with HoFH.
Source: Heart Failure Clinics - Category: Cardiology Authors: Arturo Cesaro, Fabio Fimiani, Felice Gragnano, Elisabetta Moscarella, Alessandra Schiavo, Andrea Vergara, Leo Akioyamen, Laura D ’Erasmo, Maurizio Averna, Marcello Arca, Paolo Calabrò Source Type: research