UMDF, Hope for PDCD Leaders to Brief Congressional Mitochondrial Disease Caucus on Regulatory Challenges for Rare Disease Therapies

Congressional Mitochondrial Disease Caucus briefing to explore how FDA drug review processes affect patient care and access for ultra-rare, life-threatening pediatric mitochondrial diseases, using pyruvate dehydrogenase complex deficiency (PDCD) as an example. WASHINGTON, Feb. 16, 2026...