1. Existing and emerging therapies for the treatment of familial hypercholesterolemia
- Author
-
Robert S. Rosenson
- Subjects
0301 basic medicine ,Homozygous Familial Hypercholesterolemia ,Apolipoprotein B ,HeFH, heterozygous FH ,angiopoietin-like protein 3 ,Familial hypercholesterolemia ,030204 cardiovascular system & hematology ,Biochemistry ,chemistry.chemical_compound ,0302 clinical medicine ,Endocrinology ,Medicine ,genetics ,ANGPLT3, angiopoietin-like protein 3 ,Lp(a), lipoprotein a ,biology ,familial hypercholesterolemia ,HoFH, homozygous FH ,LDLR, LDL receptor ,atherosclerotic cardiovascular disease ,Lipoprotein(a) ,Low-density lipoprotein ,lipids (amino acids, peptides, and proteins) ,ASCVD, atherosclerotic cardiovascular disease ,medicine.drug ,proprotein convertase subtilisin Kexin 9 ,medicine.medical_specialty ,Statin ,medicine.drug_class ,QD415-436 ,FH, familial hypercholesterolemia ,PCSK9, proprotein convertase subtilisin Kexin type 9 ,LDL ,03 medical and health sciences ,cholesterol-lowering therapies ,Ezetimibe ,Internal medicine ,LDLRAP1, low-density lipoprotein receptor adaptor protein 1 ,business.industry ,PCSK9 ,lipoprotein (a) ,LDL receptor ,Thematic Review Series ,Cell Biology ,medicine.disease ,CI, confidence interval ,030104 developmental biology ,chemistry ,biology.protein ,apo B, apolipoprotein B ,business - Abstract
Familial hypercholesterolemia (FH), an autosomal dominant disorder of LDL metabolism that is characterized by elevated LDL cholesterol, is commonly encountered in patients with atherosclerotic coronary heart disease. Combinations of cholesterol lowering therapies are often used to lower LDL cholesterol in patients with familial hypercholesterolemia; however, current treatment goals for LDL cholesterol are rarely achieved in patients with homozygous FH, and are difficult to achieve in patients with heterozygous FH. Therapies that lower LDL cholesterol through LDL receptor-mediated mechanisms have thus far been largely ineffective in patients with homozygous FH, particularly in those with negligible (
- Published
- 2021