Ask the Mito Doc – November 2025; Q&A
Ask the Mito Doc – November 2025 Q&A All answers today are based on personal experience of the participants. As always, please...
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Your November UMDF Newsletter: Celebrating a TK2d Therapy Approval, MELAS Survey, Mito Books, and More
Signup to receive the UMDF newsletter in your email monthly at umdf.org/newsletter-signup. FDA Approves First-Ever TK2d Therapy Earlier this...
Read moreFDA 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...
Read moreSPECIAL 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...
Read moreAsk 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...
Read morePRESS 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...
Read moreREAD THE LETTER: Leading Clinicians and Researchers Urge FDA to Reconsider Path Forward for DCA in Pyruvate Dehydrogenase Complex Deficiency
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Read moreOctober 2025 Newsletter: Celebrating an Approval, Therapeutic Pipeline News, FBXL4 Podcast, and More
Signup to receive the UMDF newsletter in your email monthly at umdf.org/newsletter-signup. FDA Announces First Approved Mito Therapy: Forzinity...
Read moreAsk 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...
Read moreFDA 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...
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