Advocacy Update – Oct 2024: FDA AdComm Brings Good News, Plus DC End of Year Business
FDA Advisory Committee Deems Potential Barth Syndrome Drug “Effective” Earlier this month, an FDA Advisory Committee voted 10-6...
Read moreFDA Advisory Committee Deems Potential Barth Syndrome Drug “Effective” Earlier this month, an FDA Advisory Committee voted 10-6...
Read moreAfter a full day of testimony that consistently demonstrated the need and efficacy of elamipretide for the treatment of Barth Syndrome, an FDA...
Read moreRead UMDF’s Comment to FDA’s Advisory Committee regarding New Drug Application for Elamipretide in Barth...
Read moreUMDF Co-Signs Letter Supporting Rare Pediatric Disease Priority Review Voucher...
Read moreIn a few weeks, we’ll join other mitochondrial disease-focused patient advocacy groups across the globe in marking World Mitochondrial Disease...
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