Related Experiment Video
Updated: Jun 21, 2025

A Familial Hypercholesterolemia Human Liver Chimeric Mouse Model Using Induced Pluripotent Stem Cell-derived Hepatocytes
Published on: September 15, 2018
Emerging therapies for refractory hypercholesterolemia: a narrative review
Agnieszka Pawlos1, Etienne Khoury2, Daniel Gaudet2
1Department of Internal Diseases & Clinical Pharmacology, Laboratory of Tissue Immunopharmacology, Medical University of Lodz, Kniaziewicza 1/5, 91-347, Lodz, Poland.
Refractory hypercholesterolemia (RH) is a condition where patients don't reach cholesterol goals despite treatment. This review explores RH therapies, emphasizing low-density lipoprotein receptor (LDLR) activity for personalized treatment strategies.
Area of Science:
- Cardiology
- Genetics
- Pharmacology
Background:
- Refractory hypercholesterolemia (RH) affects patients resistant to standard lipid-lowering treatments.
- RH significantly increases cardiovascular complication risk, particularly in familial hypercholesterolemia (FH).
- The prevalence of RH is higher than previously estimated.
Purpose of the Study:
- To provide a comprehensive overview of current and emerging therapies for RH.
- To highlight the role of low-density lipoprotein receptors (LDLRs) in RH treatment.
- To emphasize the importance of LDLR activity for personalized RH treatment.
Main Methods:
- Literature review of RH therapies.
- Analysis of genetic-based therapeutic approaches.
- Evaluation of LDLR dependency in treatment strategies.
Main Results:
- Current therapies for RH are limited, necessitating novel approaches.
- Emerging therapies, including genetic-based options, show promise.
- LDLR activity is a key determinant for therapeutic efficacy in RH.
Conclusions:
- RH requires tailored treatment strategies beyond standard therapies.
- Understanding and targeting LDLRs are crucial for effective RH management.
- Personalized medicine based on LDLR function can optimize outcomes for RH patients.
Related Concept Videos
Lipid-Lowering Drugs: Statins and Miscellaneous Agents
Treatment for Pulmonary Arterial Hypertension: Receptor Tyrosine Kinase Inhibitors and Calcium Channel Blockers
TKIs, such as imatinib (Gleevec), are particularly effective in tackling the growth and mitogenic factors that become upregulated in PAH patients. These factors contribute to the...
Heart Failure Drugs: Inhibitors of Renin-Angiotensin System
Antianginal Drugs: Calcium Channel Blockers and Ranolazine
CCBs, a diverse class that includes dihydropyridines (nifedipine) and diphenylalkylamines (verapamil and diltiazem), exert their effect by blocking calcium channels in cardiac and smooth muscle cells. This...
Targeted Cancer Therapies
There are several types of targeted therapies against...
Adrenergic Antagonists: ɑ and β-Receptor Blockers

