BOSTON, April 29, 2025--(BUSINESS WIRE)--The Barth Syndrome Foundation (BSF), the only patient advocacy organization solely dedicated to saving lives through education, advances in treatments ...

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 ...

More than 35 patients worldwide have received expanded or emergency access to elamipretide for the treatment of Barth syndrome, including many critically ill infants and toddlers affected by the ...

Barth syndrome is an ultra-rare, life threatening pediatric mitochondrial disease which is known to affect less than 150 individuals in the United States and less than 300 individuals worldwide.

No revised PDUFA target date has been communicated by the FDA. A decision on the New Drug Application for elamipretide for the treatment of Barth syndrome has been delayed again, according to ...

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.

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.

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.

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.