UMDF Connect
Ask the Mito Doc – November 2024; Q&A
Ask the Mito Doc - November 2024 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.Understanding POLG DisordersConnecting the Dots Between Common...
The Mito Fund Announces Investment in Khondrion to Support Development of One of the Most Advanced Drug Candidates for Primary Mitochondrial Disease
The United Mitochondrial Disease Foundation’s (UMDF) venture philanthropy arm, The Mito Fund, has invested in Khondrion, a Netherlands-based clinical stage biopharmaceutical company discovering and developing therapies targeting primary mitochondrial disease (PMD)....
UMDF Cosigns Letter to Congress Encouraging End-of-Year Rare Disease Legislative Priorities
UMDF Cosigns Letter to Congress Encouraging End-of-Year Action on Accelerating Kids’ Access to Care Act, Rare Pediatric Disease Priority Review Voucher Program, and More
November 2024 Newsletter: FDA News, Rare Revolution Magazine Feature, Therapeutic Pipeline Updates, and more
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Ask the Mito Doc – October 2024; Q&A
Ask the Mito Doc - October 2024 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.Connecting the Dots Between Mito and Rare DiseasesConnecting the Dots...
Advocacy Update – Oct 2024: FDA AdComm Brings Good News, Plus DC End of Year Business
FDA Advisory Committee Deems Potential Barth Syndrome Drug "Effective" Earlier this month, an FDA Advisory Committee voted 10-6 supporting approval of Stealth BioTherapeutics’ elamipretide for treating Barth syndrome, a rare mitochondrial disease that occurs mostly in...
UMDF Reacts to FDA Advisory Committee’s Elamipretide Recommendation
After a full day of testimony that consistently demonstrated the need and efficacy of elamipretide for the treatment of Barth Syndrome, an FDA advisory committee correctly recommended approval of the therapy's new drug application. The committee's decision reinforces...
UMDF’s Comment to FDA’s Advisory Committee Regarding Elamipretide
Read UMDF’s Comment to FDA’s Advisory Committee regarding New Drug Application for Elamipretide in Barth syndrome
UMDF Announces $100k Award for MEPAN Research, Bringing Total Research Grants Awarded to More Than $1 Million in the Last Year
The United Mitochondrial Disease Foundation today announced a new $100,000 grant to Madhurima Saha, PhD, of the University of Florida, for research related to Mitochondrial Enoyl CoA Reductase Protein-Associated Neurodegeneration (MEPAN), a rare mitochondrial disease...
Advocacy Update – UMDF Co-Signs Letter Supporting PRV Reauthorization
UMDF Co-Signs Letter Supporting Rare Pediatric Disease Priority Review Voucher Reauthorization