Bezafibrate as treatment in males for Barth syndrome: CARDIOMAN, a double-blind, placebo-controlled crossover RCT

Background Barth syndrome is a rare, life-threatening X-linked recessive mitochondrial disorder of lipid metabolism primarily affecting males. Previous research suggests that bezafibrate may ameliorate cellular lipid abnormalities and reduce cardiac dysfunction in an animal model. Objectives Estimat...

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Bibliographic Details

Published in:	Efficacy and Mechanism Evaluation
Main Authors:	Guido Pieles, Colin Steward, Lucy Dabner, Laura Collet, Lucy Culliford, Karen Sheehan, Lucy Ellis, Michaela Damin, Eva Sammut, Nuno Duarte, Owen Burgess, Curtis Wadey, Craig Williams, John Crosby, Sarah Groves, Aidan Searle, Borko Amulic, Chris Rice, Chiara Bucciarelli-Ducci, Andrew Ness, Julian Hamilton-Shield, Chris A Rogers, Barnaby C Reeves
Format:	Article
Language:	English
Published:	NIHR Journals Library 2024-08-01
Subjects:	randomised controlled trial barth syndrome inherited cardiomyopathy bezafibrate placebo-controlled mitochondrial metabolism metabolic rare disease crossover resveratrol
Online Access:	https://doi.org/10.3310/JDBC7982