August 29, 2025 - The Barth Syndrome Foundation (BSF) extends our sincere appreciation to FDA leadership for meeting with our community last week and hearing firsthand the devastating reality of a Barth syndrome diagnosis. “For too long, the voices of patients and families have not been adequately considered through traditional regulatory processes. We are hopeful that this time is different, and that the urgent needs of those living with the Barth syndrome will be reflected in the Agency’s decisions,” said Emily Milligan, Executive Director of BSF.

Barth syndrome remains a life-limiting condition with no FDA approved therapies and an especially high and unacceptable risk of death in early childhood. Families, patients, and BSF continue to advocate for reform that applies consistent, fair, and appropriate standards that take into account the realities of ultra-rare diseases. With FDA leadership recognizing that they have the information they need to make a decision, BSF looks forward to timely and meaningful action that ensures Americans of all ages with Barth syndrome have access to effective therapies now.