UMDF Connect
PRESS RELEASE: Leading Clinicians and Researchers Urge FDA to Reconsider Path Forward for DCA in Pyruvate Dehydrogenase Complex Deficiency
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 life-threatening mitochondrial disease WASHINGTON, D.C. — October 13, 2025 — In response to the recent Complete Response Letter...
READ THE LETTER: Leading Clinicians and Researchers Urge FDA to Reconsider Path Forward for DCA in Pyruvate Dehydrogenase Complex Deficiency
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October 2025 Newsletter: Celebrating an Approval, Therapeutic Pipeline News, FBXL4 Podcast, and More
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Ask the Mito Doc – September 2025; Q&A
Ask the Mito Doc - September 2025 Q&A All answers today are based on personal experience of the participants. As always, please consult your personal physician prior to taking any action.Topic: Seizure Management in Patients with Mitochondrial Disease ...
FDA Approves First Mitochondrial Disease Therapy: Stealth BioTherapeutics’ Elamipretide for Barth Syndrome
UMDF celebrates historic milestone for rare disease community The United Mitochondrial Disease Foundation (UMDF), the largest mitochondrial disease-focused patient advocacy group in the United States, celebrates today’s U.S. Food and Drug Administration’s approval of...
A Special World Mitochondrial Disease Week Message from UMDF President & CEO Kristen Clifford
Today marks the official kickoff of World Mitochondrial Disease Week, a time designed to help the public better understand mitochondrial disease – with the hope that increased awareness, attention, and funding will accelerate progress toward effective treatments...
UMDF Co-signs Letter Encouraging Congress to “Act Swiftly” to Reauthorize the Rare Pediatric Disease PRV Program
September 2025 Newsletter: World Mito Week, FDA News, Return of Ask The Mito Doc & Bench-to-Bedside
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UMDF Joins Other Mito Advocacy Groups to Issue Statement and Call to Action Regarding the FDA’s DCA Denial
Sept. 4, 2025 -- Earlier today, Saol Therapeutics shared the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) regarding the company’s application for dichloroacetate (DCA) to treat Pyruvate Dehydrogenase Complex Deficiency...
August 2025 Newsletter: No-Cost Genetic Testing, World Mito Week, FDA Action, Ask the Mito Doc Returns, and more
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