Latest News -Press Room

UMDF Cosigns Letter to Congress Encouraging End-of-Year Action on Accelerating Kids’ Access to Care Act, Rare Pediatric Disease Priority Review...

FDA Advisory Committee Deems Potential Barth Syndrome Drug “Effective” Earlier this month, an FDA Advisory Committee voted 10-6...

After a full day of testimony that consistently demonstrated the need and efficacy of elamipretide for the treatment of Barth Syndrome, an FDA...

Read UMDF’s Comment to FDA’s Advisory Committee regarding New Drug Application for Elamipretide in Barth...

UMDF Co-Signs Letter Supporting Rare Pediatric Disease Priority Review Voucher...

In a few weeks, we’ll join other mitochondrial disease-focused patient advocacy groups across the globe in marking World Mitochondrial Disease...