UMDF Connect
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
Ask the Mito Doc – September 2024; Q&A
Ask the Mito Doc - September 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 Genetic TestingClinical Trial Readiness: Understanding...
A special message from UMDF for World Mitochondrial Disease Week 2024
This coming week, September 16 – 22, 2024, marks World Mitochondrial Disease Week. For me, the week is deeply personal. This year marks twenty years since my infant daughter Natalie was diagnosed with mitochondrial disease. Her battle lasted only a year, but her...
Advocacy Update – UMDF Signs On to Thank You Letter to Senate Appropriations Leadership for Proposed NIH Budget Increase
UMDF Announces Two New Postdoctoral Fellowship Research Grants
On the heels of our traditional research grant funding cycle culminating at UMDF’s annual Mitochondrial Medicine conference this past June, the United Mitochondrial Disease Foundation is proud to announce the funding of two additional postdoctoral fellowship research...
Advocacy Update – Aug 2024: Help Shape the Legislative Landscape for the Mito Community Through Survey, UMDF Action Items
In a few weeks, we’ll join other mitochondrial disease-focused patient advocacy groups across the globe in marking World Mitochondrial Disease Week, September 16-22, 2024. It’s a time when we can promote understanding and recognition with the hope that increased...