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A Familial Hypercholesterolemia Human Liver Chimeric Mouse Model Using Induced Pluripotent Stem Cell-derived Hepatocytes
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Familial hypercholesterolemia - treatment update in children, systematic review

Sabina Ziółkowska1, Noëlle Kijek1, Iga Zendran1

  • 1Graduate of Wroclaw Medical University, Poland.

Pediatric Endocrinology, Diabetes, and Metabolism
|July 18, 2022
PubMed

Insights

Familial hypercholesterolaemia, a common genetic disorder, requires early diagnosis and treatment to prevent childhood symptoms and future health issues. This review updates on evolving treatment strategies for this condition.

Area of Science:

  • Cardiovascular Medicine
  • Genetics
  • Pharmacology

Background:

  • Familial hypercholesterolaemia (FH) is a prevalent genetic disorder.
  • Early onset of symptoms in childhood necessitates timely intervention.
  • Untreated FH leads to severe cardiovascular complications.

Purpose of the Study:

  • To review established and emerging treatments for familial hypercholesterolaemia.
  • To provide an updated overview of FH management strategies.
  • To highlight recent advancements in therapeutic agents for FH.

Main Methods:

  • Comprehensive literature search of recent publications.
  • Synthesis of data on existing and novel FH treatments.
  • Analysis of treatment efficacy and safety profiles.

Main Results:

  • Overview of current therapeutic options for FH.
  • Discussion of newly discovered pharmacological agents.
  • Assessment of treatment evolution and future directions.

Conclusions:

  • Early diagnosis and management of FH are crucial for preventing long-term sequelae.
  • The therapeutic landscape for FH is dynamic, with ongoing discovery of novel agents.
  • Continuous updates in treatment strategies are essential for optimal patient outcomes.

Keywords:
adolescentschildrencholesterol.hyperlipoproteinaemiahypercholesterolaemia

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