Therapeutic Options for Homozygous Familial Hypercholesterolemia: The Role of Lomitapide.

Author: AvernaMaurizio R, CefalùAngelo B, GiammancoAntonina, NotoDavide

Paper Details 
Original Abstract of the Article :
Lomitapide (Juxtapid® in US and Lojuxta® in Europe) is the first developed inhibitor of the Microsomal Triglyceride Transfer Protein (MTP) approved as a novel drug for the management of Homozygous Familial Hypercholesterolemia (HoFH). It acts by binding directly and selectively to MTP thus decreasin...See full text at original site
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引用元:
https://doi.org/10.2174/0929867326666190121120735

データ提供:米国国立医学図書館(NLM)

Targeting the Microsomal Triglyceride Transfer Protein

This research delves into the realm of [cardiovascular disease] and explores innovative approaches to treating [homozygous familial hypercholesterolemia (HoFH)]. The study focuses on [lomitapide], a novel drug that inhibits the [Microsomal Triglyceride Transfer Protein (MTP)]. The authors investigated the mechanism of action of lomitapide and its efficacy in managing HoFH. They found that lomitapide effectively [decreases the assembly and secretion of apo-B containing lipoproteins] in both the liver and intestine, providing a potential therapeutic option for patients with this rare and debilitating condition.

Lomitapide's Potential in HoFH Management

The study reveals that [lomitapide] is a promising therapeutic agent for HoFH, a condition characterized by extremely high cholesterol levels. By inhibiting MTP, lomitapide effectively [reduces the production and secretion of harmful lipoproteins] which can lead to severe cardiovascular complications. This discovery offers hope for improving the health and well-being of patients with HoFH.

HoFH and Cardiovascular Health

HoFH is a serious condition that can significantly increase the risk of [heart attacks, strokes, and premature death]. Understanding the role of MTP in cholesterol metabolism is crucial for developing effective treatments for this disease. The development of drugs like lomitapide provides a new avenue for tackling this challenge.

Dr.Camel's Conclusion

This research reminds us that even in the vast desert of medical challenges, we can find oases of hope. The development of lomitapide signifies a significant advancement in the treatment of HoFH, offering patients a new path towards better health and a brighter future. This study underscores the importance of scientific innovation in addressing complex medical conditions and improving the lives of patients worldwide.

Date :
  1. Date Completed 2020-07-13
  2. Date Revised 2020-07-13
Further Info :

Pubmed ID

30663562

DOI: Digital Object Identifier

10.2174/0929867326666190121120735

Related Literature

SNS
PICO Info
in preparation
Languages

English

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