Related Experiment Video
Updated: Feb 14, 2026
10:56
A Familial Hypercholesterolemia Human Liver Chimeric Mouse Model Using Induced Pluripotent Stem Cell-derived Hepatocytes
Published on: September 15, 2018
8.5K
New Therapeutic Approaches for Familial Hypercholesterolemia
1Departments of Medicine and Genetics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania 19104; email: rader@mail.med.upenn.edu , eajufo@gmail.com.
Annual Review of Medicine
|February 8, 2018
Summary
Familial hypercholesterolemia (FH) is a genetic condition causing high LDL-C. Four new agents approved from 2012-2015 offer improved LDL-C lowering for FH patients, with more agents in development.
Area of Science:
- Cardiology
- Genetics
- Pharmacology
Background:
- Familial hypercholesterolemia (FH) is a prevalent genetic disorder.
- It leads to elevated low-density lipoprotein cholesterol (LDL-C) and premature cardiovascular disease.
- Current therapies show limitations in managing FH effectively.
Purpose of the Study:
- To review novel LDL-C-lowering agents approved for FH.
- To discuss agents currently in clinical development for FH.
- To focus on mechanisms, efficacy, and safety profiles.
Main Methods:
- Review of FDA-approved LDL-C-lowering agents (2012-2015).
- Analysis of agents in clinical development for FH.
- Examination of scientific literature on efficacy and safety.
Main Results:
- Four novel agents were approved for FH between 2012 and 2015.
- These agents demonstrate significant LDL-C reduction in FH cohorts.
- Ongoing research is exploring additional promising therapies.
Conclusions:
- Novel therapeutic agents offer improved management for Familial hypercholesterolemia.
- Understanding mechanisms, efficacy, and safety is crucial for FH treatment.
- Continued development is vital to address unmet needs in FH care.
Related Concept Videos
02:47Protein Families
17.2K
Protein families are groups of homologous proteins; that is, they have similarities in amino acid sequences and three-dimensional structures. Protein families usually occur because of gene duplication, where an additional copy of a gene is inserted into the genome of an organism. Mutations that change the amino acids but still allow the protein to be properly synthesized, will lead to new protein family members. If these new proteins contain similar amino acids in key...
17.2K
02:47Protein Families
4.5K
4.5K
01:57Gene Families
10.0K
Gene families consist of groups of genes proposed to have originated from a common ancestor. Typically these arise through events in which a gene or genes are mistakenly duplicated during cell division. Unlike their parent genes (which are subject to selection pressure to maintain function), these gene copies do not need to preserve their sequences and may evolve at a relatively faster rate.
Occasionally these regions can be adapted to take on new roles within the organism, becoming novel genes...
Occasionally these regions can be adapted to take on new roles within the organism, becoming novel genes...
10.0K
01:57Gene Families
3.9K
3.9K
01:30Family Therapy
620
Family therapy conceptualizes psychological challenges as arising from dysfunctional interactions within the family unit, rather than as isolated issues within individuals. This approach seeks to address and transform the patterns of communication, roles, and relationships within families to promote healthier dynamics and emotional well-being for all members.
Strategic Family Therapy
Strategic family therapy emphasizes resolving communication barriers and improving problem-solving abilities...
Strategic Family Therapy
Strategic family therapy emphasizes resolving communication barriers and improving problem-solving abilities...
620
01:18Sources of Self-Esteem I: Family Experience
197
Self-esteem, a crucial component of psychological development, is significantly shaped by familial experiences. The early parent-child relationship serves as a foundational influence on a child's self-concept, with long-lasting effects extending into adolescence and adulthood.Parental Behaviors and Early Self-Esteem FormationEmpirical studies have identified four principal parental behaviors that foster healthy self-esteem in children. These include expressions of acceptance, affection, and...
197