Novel Therapies for Treating Familial Hypercholesterolemia

  title={Novel Therapies for Treating Familial Hypercholesterolemia},
  author={Salman Jamaluddin Bandeali and Jad Daye and Salim S. Virani},
  journal={Current Atherosclerosis Reports},
Familial hypercholesterolemia is an inherited disorder associated with early accelerated atherosclerosis with morbidity and mortality resulting from premature cardiovascular disease. Affected individuals have extreme elevations in low-density lipoprotein cholesterol levels. Patients usually do not achieve target reductions in cholesterol levels with conventional antihyperlipidemic pharmacotherapy. This unmet need has resulted in the recent development and approval of novel therapies targeting… 
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Management of familial hypercholesterolemia: a review of the recommendations from the National Lipid Association Expert Panel on Familial Hypercholesterolemia.
  • J. Robinson
  • Medicine, Biology
    Journal of managed care pharmacy : JMCP
  • 2013
Recommendations for the screening and treatment of patients with Familial Hypercholesterolemia include mipomersen, which was approved by the FDA in January 2013 and received FDA approval for use only in patients with HoFH, and novel treatments currently in development include PCSK9 inhibitors.
Homozygous familial hypercholesterolemia: current perspectives on diagnosis and treatment.
Molecular genetics of the LDL receptor gene in familial hypercholesterolemia
79 additional mutations are described and the insights that all 150 mutations have provided into the structure/function relationship of the receptor protein and the clinical manifestations of FH are reviewed.
Monogenic hypercholesterolemia: new insights in pathogenesis and treatment.
A subset of patients with very high plasma LDL-C levels have monogenic forms of hyper-cholesterolemia, which are associated with the deposition of cholesterol in tissues, producing xanthomas and coronary atherosclerosis, and how insights gleaned from the study of these disorders may be extended to the treatment of hypercholesterine in the general population is discussed.
Novel nonstatin strategies to lower low-density lipoprotein cholesterol
There remains an unmet need to reduce elevated low-density lipoprotein cholesterol (LDL-C) in patients who are maximized on current therapy or intolerant to statins. Several novel agents have been
Long-term improvement of hypercholesterolemia after ex vivo gene therapy in LDLR-deficient rabbits.
An animal model for FH, the Watanabe Heritable Hyperlipidemic rabbit, was used to develop an approach for liver-directed gene therapy based on transplantation of autologous hepatocytes that were genetically corrected ex vivo with recombinant retroviruses.
Apolipoprotein B Synthesis Inhibition With Mipomersen in Heterozygous Familial Hypercholesterolemia: Results of a Randomized, Double-Blind, Placebo-Controlled Trial to Assess Efficacy and Safety as Add-On Therapy in Patients With Coronary Artery Disease
Mipomersen is an effective therapy to further reduce apolipoprotein B–containing lipoproteins, including LDL and lipoprotein(a), in HeFH patients with coronary artery disease on statins and other lipid-lowering therapy.