Related Experiment Video

Updated: Dec 5, 2025

A Familial Hypercholesterolemia Human Liver Chimeric Mouse Model Using Induced Pluripotent Stem Cell-derived Hepatocytes
10:56

A Familial Hypercholesterolemia Human Liver Chimeric Mouse Model Using Induced Pluripotent Stem Cell-derived Hepatocytes

Published on: September 15, 2018

8.4K

Treatment of heterozygous familial hypercholesterolemia: what does the future hold?

Georgios Polychronopoulos1, Konstantinos Tziomalos1

  • 1First Propedeutic Department of Internal Medicine, Medical School, Aristotle University of Thessaloniki, AHEPA Hospital , Thessaloniki, Greece.

Expert Review of Clinical Pharmacology
|October 19, 2020
PubMed

Insights

New lipid-lowering agents inclisiran and bempedoic acid show promise for managing heterozygous familial hypercholesterolemia (heFH). Further research is needed to confirm their impact on cardiovascular health and cost-effectiveness.

Area of Science:

  • Cardiovascular Medicine
  • Metabolic Disorders
  • Pharmacology

Background:

  • Heterozygous familial hypercholesterolemia (heFH) is a prevalent genetic condition increasing cardiovascular risk.
  • Current lipid-lowering therapies often fail to achieve target low-density lipoprotein cholesterol (LDL-C) levels in a significant number of heFH patients.

Purpose of the Study:

  • To review the safety and efficacy of novel LDL-C lowering agents, inclisiran and bempedoic acid.
  • To discuss their potential role in managing patients with heterozygous familial hypercholesterolemia (heFH).

Main Methods:

  • Comprehensive literature search of the PubMed database up to August 2020.
  • Review and synthesis of data on the efficacy and safety of inclisiran and bempedoic acid.

Main Results:

  • Inclisiran demonstrates a prolonged lipid-lowering effect, potentially improving treatment adherence in heFH.
  • Bempedoic acid offers additional LDL-C reduction in patients on high-intensity statins, with oral administration as a potential advantage.

Conclusions:

  • Inclisiran and bempedoic acid represent potential advancements in heFH management.
  • Further evaluation of their impact on cardiovascular morbidity and cost-effectiveness is crucial before widespread clinical adoption.
Abstract

Keywords:
Bempedoic acidfamilial hypercholesterolemiainclisiran

More Related Videos

LDL Cholesterol Uptake Assay Using Live Cell Imaging Analysis with Cell Health Monitoring08:45

LDL Cholesterol Uptake Assay Using Live Cell Imaging Analysis with Cell Health Monitoring

Published on: November 17, 2018

13.9K
Isolation and Analysis of Plasma Lipoproteins by Ultracentrifugation06:47

Isolation and Analysis of Plasma Lipoproteins by Ultracentrifugation

Published on: January 28, 2021

12.1K

Related Concept Videos

Atherosclerosis III: Management01:26

Atherosclerosis III: Management

Management of atherosclerosis involves an integrated strategy encompassing pharmacological treatment, surgical interventions, lifestyle changes, and nutrition therapy to address the multifactorial nature of the disease.Pharmacological TherapyA cornerstone of atherosclerosis management is the use of pharmacological agents. Statins, such as atorvastatin, are pivotal in inhibiting HMG-CoA reductase, an enzyme that catalyzes an initial step in cholesterol synthesis in the liver. This reduction in...
219
Lipid-Lowering Drugs: Statins and Miscellaneous Agents01:20

Lipid-Lowering Drugs: Statins and Miscellaneous Agents

Hyperlipidemia, a medical condition often referred to as high cholesterol, is characterized by abnormally elevated levels of lipids in the bloodstream. When present in excess, these lipids, specifically cholesterol and triglycerides, can lead to serious health complications, often involving cardiovascular diseases. Illnesses like atherosclerosis, heart attacks, and pancreatitis have all been linked to untreated hyperlipidemia. This means controlling and regulating cholesterol and triglyceride...
1.2K
Cholesterol: Significance and Regulation01:29

Cholesterol: Significance and Regulation

Although not a source of energy, cholesterol plays a significant role as a foundational structure for bile salts, steroid hormones, and vitamin D, as well as being a crucial component of plasma membranes. Approximately 15% of blood cholesterol is derived from our diet, with the remainder synthesized from acetyl CoA by the liver and intestines. Cholesterol is eliminated from the body through its conversion into bile salts, which are eventually discarded in the feces.
Considering cholesterol and...
1.1K
Treatment for Pulmonary Arterial Hypertension: Endothelin Receptor Antagonists01:18

Treatment for Pulmonary Arterial Hypertension: Endothelin Receptor Antagonists

Endothelins (ETs) are potent vasoactive peptides critical in the human body's various physiological and pathological processes. One of the most promising therapeutic strategies for treating pulmonary arterial hypertension (PAH) involves counteracting the effects of these endothelins using a class of drugs known as endothelin receptor antagonists.
ETs are synthesized through a complex sequence of enzymatic steps, primarily involving an enzyme referred to as endothelin-converting enzyme...
309
Treatment for Pulmonary Arterial Hypertension: Receptor Tyrosine Kinase Inhibitors and Calcium Channel Blockers01:26

Treatment for Pulmonary Arterial Hypertension: Receptor Tyrosine Kinase Inhibitors and Calcium Channel Blockers

Receptor tyrosine kinase inhibitors (TKIs) and calcium channel blockers (CCBs) are two critical categories of drugs employed in the treatment of pulmonary artery hypertension (PAH). PAH is a disease that causes high blood pressure in the pulmonary arteries, resulting in chest pain, fatigue, and shortness of breath.
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...
345
Treatment for Pulmonary Arterial Hypertension: Phosphodiesterase Inhibitors01:28

Treatment for Pulmonary Arterial Hypertension: Phosphodiesterase Inhibitors

Phosphodiesterase 5 (PDE5) inhibitors are potent enzymes that function to hydrolyze cyclic nucleotides to their corresponding 5' monophosphates. Their unique biochemical properties have been applied in treating Pulmonary Arterial Hypertension (PAH).
Among the PDE5 inhibitors, sildenafil (Revatio) stands out as a competitive and selective inhibitor. It operates by elevating cellular levels of cGMP and augmenting signaling through the cGMP-PKG pathway, promoting vasodilation. Upon oral...
369