Elamipretide, a cell-permeable peptide compound, is designed to improve mitochondrial function by targeting the inner mitochondrial membrane where it binds reversibly to cardiolipin.

Stealth Biotherapeutics Inc., and, more importantly, patients with Barth syndrome, faced another disappointing delay April 29 when the U.S. FDA kicked its approval decision down the road for Stealth’s ...

Today, I worry it could come too late to help my son. My son, Declan, has Barth syndrome. This ultra-rare genetic mitochondrial disease affects fewer than 150 people in the United States.