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...
Read: FDA Approves First-Ever TK2d TherapyLatest News -Press Room
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...
Read: SPECIAL NATIONAL AUTHOR’S DAY FEATURE: Mito Patient and His Mom Talk About Self-Publishing “Children’s Chapter Adventure” BookPRESS 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: PRESS RELEASE: Leading Clinicians and Researchers Urge FDA to Reconsider Path Forward for DCA in Pyruvate Dehydrogenase Complex DeficiencyOctober 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: October 2025 Newsletter: Celebrating an Approval, Therapeutic Pipeline News, FBXL4 Podcast, and 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...
Read: FDA Approves First Mitochondrial Disease Therapy: Stealth BioTherapeutics’ Elamipretide for Barth SyndromeA 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...
Read: A Special World Mitochondrial Disease Week Message from UMDF President & CEO Kristen CliffordSeptember 2025 Newsletter: World Mito Week, FDA News, Return of Ask The Mito Doc & Bench-to-Bedside
Signup 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-BedsideUMDF 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...
Read: UMDF Joins Other Mito Advocacy Groups to Issue Statement and Call to Action Regarding the FDA’s DCA DenialAugust 2025 Newsletter: No-Cost Genetic Testing, World Mito Week, FDA Action, Ask the Mito Doc Returns, and more
Signup 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 more