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Updated: May 5, 2026
10:56
A Familial Hypercholesterolemia Human Liver Chimeric Mouse Model Using Induced Pluripotent Stem Cell-derived Hepatocytes
Published on: September 15, 2018
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Novel therapies for treating familial hypercholesterolemia
Salman J Bandeali1, Jad Daye, Salim S Virani
1Section of Cardiology, Department of Medicine, Texas Heart Institute, St. Luke's Episcopal Hospital, 6720 Bertner, MC 1-133, Houston, TX, 77030, USA, salmanbandeali@hotmail.com.
Current Atherosclerosis Reports
|December 3, 2013
Summary
Familial hypercholesterolemia causes early cardiovascular disease due to high LDL cholesterol. New therapies offer improved management beyond traditional treatments for this inherited condition.
Area of Science:
- Cardiology
- Genetics
- Pharmacology
Background:
- Familial hypercholesterolemia (FH) is an inherited disorder leading to premature atherosclerosis and cardiovascular disease.
- Individuals with FH exhibit extremely elevated low-density lipoprotein cholesterol (LDL-C) levels.
- Conventional antihyperlipidemic medications often fail to achieve target LDL-C reductions in FH patients.
Purpose of the Study:
- To provide a concise overview of familial hypercholesterolemia.
- To discuss novel pharmacotherapies for managing FH.
- To address the unmet need in FH treatment.
Main Methods:
- Literature review of familial hypercholesterolemia.
- Summary of current understanding of FH pathophysiology.
- Discussion of recently approved and emerging pharmacotherapies targeting cholesterol pathways.
Main Results:
- FH is characterized by genetic defects leading to severe hypercholesterolemia.
- Novel therapies target various pathways, including PCSK9 inhibition and others.
- These newer agents offer alternative or adjunctive treatment options.
Conclusions:
- Familial hypercholesterolemia requires effective management strategies due to high cardiovascular risk.
- Novel pharmacotherapies represent a significant advancement in treating FH.
- Further research and clinical application of these therapies are crucial for improving patient outcomes.
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