Results 211 to 220 of about 41,331 (231)

A phase 2/3 randomized clinical trial followed by an open-label extension to evaluate the effectiveness of elamipretide in Barth syndrome, a genetic disorder of mitochondrial cardiolipin metabolism

Genetics in Medicine, 2021
Brittany Decroes, Hilary J Vernon
exaly  

AAV Gene Therapy Prevents and Reverses Heart Failure in a Murine Knockout Model of Barth Syndrome

Circulation Research, 2020
Suya Wang, Yifei Li, Yang Xu
exaly  

Barth syndrome: cardiolipin, cellular pathophysiology, management, and novel therapeutic targets

Molecular and Cellular Biochemistry, 2021
Grant M Hatch
exaly  

Clinical presentation and natural history of Barth Syndrome: An overview

Journal of Inherited Metabolic Disease, 2022
Carolyn Taylor, Brittany Decroes, Hilary J Vernon   +2 more
exaly  

Modeling the mitochondrial cardiomyopathy of Barth syndrome with induced pluripotent stem cell and heart-on-chip technologies

Nature Medicine, 2014
Megan L Mccain, Luhan Yang, Aibin He
exaly  

Cardiac and Skeletal Muscle Defects in a Mouse Model of Human Barth Syndrome

Journal of Biological Chemistry, 2011
Frédéric M Vaz, Riekelt H Houtkooper, Janaka Wansapura   +2 more
exaly  

Barth syndrome: Cellular compensation of mitochondrial dysfunction and apoptosis inhibition due to changes in cardiolipin remodeling linked to tafazzin (TAZ) gene mutation

Biochimica Et Biophysica Acta - Molecular Basis of Disease, 2013
Guillaume Vial, Riekelt H Houtkooper, Eyal Gottlieb   +2 more
exaly  

Barth syndrome associated with triple mutation

Pediatrics International, 2018
Nobuyuki Tsujii, Takeharu Hayashi, Tamaki Hayashi, Akinori Kimura, Toshiya Nishikubo   +4 more
openaire   +2 more sources

Barth syndrome, a human disorder of cardiolipin metabolism

FEBS Letters, 2006
Michael Schlame
exaly  

Cardiac‐specific succinate dehydrogenase deficiency in Barth syndrome

EMBO Molecular Medicine, 2016
Sylvie Callegari, Karl Toischer, Britta Brügger   +2 more
exaly