Corinne Blancard

Ebselen restores mitochondrial function through metabolic rewiring in Barth syndrome and related mitochondrial diseases.

Despite major advances in the genetic diagnosis of mitochondrial disorders, effective disease-modifying therapies remain scarce. Here, we identify ebselen (EBS) as a promising therapeutic candidate through a phenotypic drug screen in a yeast model of Barth syndrome, a cardiomyopathy caused by defective cardiolipin matu...