(Sept. 23, 2023) As we wind down the final days of World Mitochondrial Disease Week, we want to take a moment to thank the UMDF advocates who helped us have over 100 touchpoints with Congress this Wednesday on UMDF’s Virtual Capitol Hill Day.
Thanks to your passionate words, our community was heard loud and clear this week.
Legislators heard how mito families are paying hundreds – sometimes thousands – of dollars a month on vitamins, supplements, and nutrition simply to live and why the Medical Nutrition Equity Act could help lighten the financial burden.
They heard the barriers mitochondrial disease patients face when they receive care from out-of-state medical specialists, and how the Accelerating Kids’ Access to Care Act could help eliminate red tape in getting world-class care for young mito patients.
House members heard the desperate need for representatives to be the mitochondrial disease community’s voice on Capitol Hill and were asked to join the Mitochondrial Disease Caucus.
And finally, they heard deeply personal stories from our community. Of hope. Of heartache. Of the lengths families go to ensure patients live to see the day there is a treatment, or maybe even a cure, for their disease.
In an increasingly polarized world, it’s easy to become jaded with the government. But never doubt the power of your words. It’s only through your words and stories that we accomplish great things.
Your words are why the Department of Defense is awarding $5-$10 million annually for mitochondrial disease research — and why UMDF is invited to submit names for potential peer reviewers every year for that research. Your words are why the telehealth expansion remains in place today. And your words are why we have a Mitochondrial Disease Caucus in the House of Representatives in the first place.
On behalf of the entire mitochondrial disease community, we thank those you bravely shared your stories with Congress.
For those who were not part of Capitol Hill Day, there is still time for your voice to be heard. On the UMDF Advocacy Action Center, with a simple click of a few buttons, you can encourage your members of congress to act on the Medical Nutrition Equity Act or request your House member to join the Mitochondrial Disease Caucus. If you prefer a more personal touch, feel free to reach out to UMDF’s Advocacy Director for bullet points on important mito-related legislation.
Sign a Petition to Support an FDA Review of Elamipretide in Barth Syndrome
In partnership with our friends at the Barth Syndrome Foundation, UMDF encourages you to sign a petition asking for “a fair, equitable and appropriate review” of elamipretide in Barth syndrome by the FDA. Clinical trials for elamipretide, the first-ever potential drug for Barth syndrome, started over five years ago. Families credit the drug with significant improvements in the health, strength, and energy of patients, but have run into roadblocks with FDA approval. Like most mitochondrial diseases, Barth syndrome is an ultra-rare disease that makes clinical trial data harder to review due to the smaller sample size. Congress has authorized the FDA to use flexibility in these cases, but the FDA has thus far refused to exercise this flexibility. Sign the petition to encourage a review today.