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.
The Barth Syndrome Foundation (BSF) acknowledges Stealth BioTherapeutics’ (Stealth) announcement today that it has resubmitted its third New Drug Application (NDA) for elamipretide, the only therapy in late-stage development for Barth syndrome, a devastating, ultra-rare, mitochondrial disease.
Barth Syndrome Foundation is a global facilitator for advancing understanding of and developing treatments for Barth syndrome.
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