The FDA has communicated to Stealth BioTherapeutics that it would not meet elamipretide’s PDUFA target date for the treatment of Barth syndrome.

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.

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.

Clinical studies show elamipretide can improve mitochondria function and boost muscle strength by 45% and heart function by 40% in patients with Barth syndrome. Most patients from the original ...

Barth syndrome is an ultra-rare genetic mitochondrial disease leading to exercise intolerance, muscle weakness, debilitating fatigue, heart failure, recurrent infections, and delayed growth.