Latest News -Press Room

With the recent election results, we’ve been getting some inquiries about the impact of a second Trump Administration on mitochondrial disease...

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...