The Barth Syndrome Foundation (BSF), the only patient advocacy organization solely dedicated to saving lives through education, advances in treatments, and finding a cure for Barth syndrome, today ...

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 ...

The U.S. Food and Drug Administration has delayed its decision on marketing approval for Stealth BioTherapeutics' treatment for a rare genetic condition that typically affects boys, the drug developer ...

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

Moreover, the agency did not indicate when it may now complete its review for the drug, which is called elamipretide and was developed to combat Barth syndrome.

This therapy improved his heart function, energy levels and overall ... to support the approval of elamipretide for treating Barth syndrome. This was tremendous progress for our small community ...

This therapy improved his heart function, energy levels and overall ... to support the approval of elamipretide for treating Barth syndrome. This was tremendous progress for our small community ...

The Needham, Massachusetts-based company's lead drug, elamipretide, is a potential treatment for Barth syndrome - a condition that affects the heart, muscles, immune system and delays growth.