Recent studies have shed light on the critical role of cardiolipin in mitochondrial function and how its deficiency can lead to severe health issues in Barth syndrome. For instance, one study ...

The FDA has extended the review period for the New Drug Application for elamipretide for the treatment of Barth syndrome.

If approved, this would be the first marketing authorization for elamipretide, a first-in-class mitochondria-targeted therapeutic, and the first FDA-approved therapy for Barth syndrome.

If approved, this would be the first marketing authorization for elamipretide, a first-in-class mitochondria-targeted therapeutic, and the first FDA-approved therapy for Barth syndrome. About Barth ...