Leading advocacy groups and more than 80 scientific and medical leaders call on FDA to act urgently on DCA approval for PDCD, a rare and...
Read: PRESS RELEASE: Leading Clinicians and Researchers Urge FDA to Reconsider Path Forward for DCA in Pyruvate Dehydrogenase Complex DeficiencySignup to receive the UMDF newsletter in your email monthly at umdf.org/newsletter-signup. FDA Announces First Approved Mito Therapy: Forzinity...
Read: October 2025 Newsletter: Celebrating an Approval, Therapeutic Pipeline News, FBXL4 Podcast, and MoreUMDF celebrates historic milestone for rare disease community The United Mitochondrial Disease Foundation (UMDF), the largest mitochondrial...
Read: FDA Approves First Mitochondrial Disease Therapy: Stealth BioTherapeutics’ Elamipretide for Barth SyndromeToday marks the official kickoff of World Mitochondrial Disease Week, a time designed to help the public better understand mitochondrial...
Read: A Special World Mitochondrial Disease Week Message from UMDF President & CEO Kristen CliffordSignup to receive the UMDF newsletter in your email monthly at umdf.org/newsletter-signup. UMDF Preps for Mitochondrial Disease Awareness Week...
Read: September 2025 Newsletter: World Mito Week, FDA News, Return of Ask The Mito Doc & Bench-to-BedsideSept. 4, 2025 — Earlier today, Saol Therapeutics shared the U.S. Food and Drug Administration (FDA) has issued a Complete Response...
Read: UMDF Joins Other Mito Advocacy Groups to Issue Statement and Call to Action Regarding the FDA’s DCA DenialSignup to receive the UMDF newsletter in your email monthly at umdf.org/newsletter-signup. ...
Read: August 2025 Newsletter: No-Cost Genetic Testing, World Mito Week, FDA Action, Ask the Mito Doc Returns, and moreWe’re excited to unite with other mitochondrial disease-focused patient advocacy groups across the globe to spread awareness on World...
Read: Energize the Fight – World Mitochondrial Disease Week 2025Barth Syndrome Foundation, UMDF, and MitoAction leadership were recently notified by Stealth BioTherapeutics that FDA has refused their request...
Read: Advocacy Action: Ask FDA to Reconsider Elamipretide Decision