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: Estim...

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Publicado en:	Efficacy & Mechanism Evaluation (EME) Vol. 11; no. 13; pp. 1 - 88
Autores principales:	Pieles, Guido, Steward, Colin, Dabner, Lucy, Collet, Laura, Culliford, Lucy, Sheehan, Karen, Ellis, Lucy, Damin, Michaela, Sammut, Eva, Duarte, Nuno, Burgess, Owen, Wadey, Curtis, Williams, Craig, Crosby, John, Groves, Sarah, Searle, Aidan, Amulic, Borko, Rice, Chris, Bucciarelli-Ducci, Chiara, Ness, Andrew
Formato:	research tables/charts randomized controlled trial Journal Article
Publicado:	NIHR Journals Library Aug2024
Acceso en línea:	Ver este registro en EBSCOhost