Novel therapeutic opportunities for familial lecithin:cholesterol acyltransferase deficiency: promises and challenges.

doi:10.1097/MOL.0000000000000864

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Article

Novel therapeutic opportunities for familial lecithin:cholesterol acyltransferase deficiency: promises and challenges.

Current opinion in lipidology - 1 Apr 2023

Vitali Cecilia, Rader Daniel J, Cuchel Marina

Abstract excerpt

PURPOSE OF REVIEW: Genetic lecithin:cholesterol acyltransferase (LCAT) deficiency is a rare, inherited, recessive disease, which manifests as two different syndromes: Familial LCAT deficiency (FLD) and Fish-eye disease (FED), characterized by low HDL-C and corneal opacity. FLD patients also develop anaemia and renal disease. There is currently no therapy for FLD, but novel therapeutics are at different stages of...

Topics

Humans
Biomarkers
Disease Progression
Kidney
Kidney Diseases
Lecithin Cholesterol Acyltransferase Deficiency
Phenotype
Phosphatidylcholine-Sterol O-Acyltransferase

Source

Open in PubMed

Identifiers

PMID: 36473023
DOI: 10.1097/MOL.0000000000000864

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 31 Aug 2026.

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