The Barth Syndrome Foundation (BSF), the only patient advocacy organization dedicated to Barth syndrome and saving lives around the world through education, advances in treatment and finding a cure, today announced that it is petitioning the U.S. Food and Drug Administration (FDA) to review the New Drug Application (NDA) for elamipretide, the only potential treatment for Barth syndrome, in a fair, equitable and appropriate manner. Despite compelling evidence that elamipretide has been well-tolerated and has demonstrated clinical benefit in Barth syndrome, and despite ongoing efforts by the Barth Syndrome Foundation and other advocates to engage with regulators, the FDA has refused to review an NDA to date. The petition ran from mid-September to mid-November, and in just two months, garnered nearly 20,000 signatures from individuals across all 50 U.S. states, plus Washington, DC, Puerto Rico, the U.S. Virgin Islands and deployed military personnel.

You can read the full press release here.