UMDF Connect
Your November UMDF Newsletter: Celebrating a TK2d Therapy Approval, MELAS Survey, Mito Books, and More
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FDA Approves First-Ever TK2d Therapy
11/3/25, 5pm EST -- Earlier today, the U.S. Food and Drug Administration (FDA) approved UCB’s Kygevvi (doxecitine and doxribtimine) to treat the mitochondrial disease thymidine kinase 2 deficiency (TK2d). “It’s hard to put into words what today’s decision means for...
SPECIAL NATIONAL AUTHOR’S DAY FEATURE: Mito Patient and His Mom Talk About Self-Publishing “Children’s Chapter Adventure” Book
KSS Patient and Mom Talk About Self-Publishing Their "Children's Chapter Adventure" Book Thomas Gordon is not your typical author. Diagnosed with the mitochondrial disease Kearns-Sayre syndrome in 2014, he lives life totally blind, relies on hearing aids, and cannot...
Ask the Mito Doc – October 2025; Q&A
Ask the Mito Doc - October 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: Fact or Fiction: Separating Science from Speculation in Mitochondrial...
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...