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

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...

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...

Read UMDF’s Comment to FDA’s Advisory Committee regarding New Drug Application for Elamipretide in Barth syndrome

UMDF Co-Signs Letter Supporting Rare Pediatric Disease Priority Review Voucher Reauthorization

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...

UMDF Issues Response to Congressional 21st Century Cures Act RFIDOWNLOAD this document (PDF) >>

As part of our commitment to raising patient voices in the regulatory process, we wanted to share an invite from the FDA's Center for Biologics Evaluation and Research (CBER), which is hosting a patient and care partner listening meeting – Patient and Care Partner...

Last week, UMDF hosted a congressional briefing with the U.S. House of Representatives’ Mitochondrial Disease Caucus, which featured speakers from across the mito community, including Caucus Co-chairs Rep. Brian Fitzpatrick & Rep. Jim McGovern; Andy Dearth,...

Nearly a decade ago, thanks to UMDF advocates, mitochondrial disease became an eligible topic in the Department of Defense’s Peer Reviewed Medical Research Program (PRMRP). The program has since provided more than $70 million in federal funds for mitochondrial disease...

Rare Disease Day On February 29, we'll join the nearly 300 million affected patients and their loved ones across the world in marking Rare Disease Day. It's a day where we collectively raise awareness and generate support for those living with rare disease -- but...

In a landmark move, in December 2023, the U.S. Food and Drug Administration (FDA) released a detailed guidance document titled “Rare Diseases: Considerations for the Development of Drugs and Biological Products.” The FDA guidance document is a significant stride...

UMDF Advocacy Update – Dec. 2023 The House is scheduled to be out of session until January, while the Senate is scheduled to remain in session this week to continue to work toward an agreement on funding for Ukraine, Israel, and US border reform, as well as...

Published 11/29/23 President Biden Signs Funding Bill, Averting a Government Shutdown On November 16, President Biden signed the stopgap funding bill passed by the House and Senate. The short-term two-tiered spending bill includes temporary extensions of expiring...

Published 10/24/23; Updated 10/25/23, 4:30 pm ET The wait for a new House of Representatives speaker has ended with Rep. Mike Johnson of Louisiana. With that business handled, looming large is the emergency supplemental spending package for Israel and Ukraine, which...

(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...

UMDF Washington Update – May 25, 2023  Debt Limit Negotiations Continue   House Speaker Kevin McCarthy (R-Calif.) met with President Joe Biden on May 22, 2023, and had a "productive discussion," but they have not yet reached a deal on raising the U.S. debt ceiling....

Congress recently returned from its two-week spring recess, kicking off a busy work period leading to the Memorial Day recess. This week, four House committees held health-related hearings, and the Senate HELP Committee is expected soon to take up Pharmacy Benefit...

Every year, UMDF, with help from the Congressional Mitochondrial Disease Caucus (co-chaired by Reps. Fitzpatrick (R-PA) and McGovern (D-MA) work to educate Congress on federal funding priorities for mitochondrial disease. Specifically, we advocate...

For many years, UMDF has worked to encourage greater mitochondrial disease research funding through the National Institutes of Health (NIH). And we have also worked to direct mitochondrial disease research funding through the Peer Reviewed Medical Research Program of...

Year-End Advocacy Success Recap 2022 ended well for UMDF, thanks to the activism of UMDF members, with the final federal funding bill passed and signed by President Biden on December 29th. As mentioned last month, the bill included Department of Defense (DoD) funding...

On Tuesday, December 20, 2022, lawmakers unveiled their long-awaited $1.7 trillion government funding package. The package includes 12 appropriations bills, including the Defense Appropriations bill. Thanks to our advocacy efforts, mitochondrial disease continues to...

This week, as part of World Mitochondrial Disease Week, UMDF President & CEO Brian Harman caught up with the offices of several members of Congress to talk about mitochondrial disease and the support we need from the federal government. We wrapped up each call...

This coming week, September 18 – 24, marks World Mitochondrial Disease Week. It provides a time for the entire mito community to share our joys, our sorrows, our hopes, and our fears. It’s a week when we can reflect on all we’ve collectively accomplished, while...

White House Releases Fiscal Year (FY) 2023 Budget Request - Big Proposed Increases for Research On Monday, March 28, 2022, President Biden released his proposed fiscal year (FY) 2023 budget. The president’s budget request starts the Congressional budget process. The...

Omnibus Bill Includes 151-Day Extension of Telehealth Waivers Earlier this week, President Biden signed a bill to fund the government through September (the fiscal year 2022) and send humanitarian aid to Ukraine.  The bill also included several health priorities,...

United Mitochondrial Disease Foundation 2022 Congressional Outlook As it reconvenes, Congress faces a packed agenda in a legislative year that will be shortened by the upcoming midterm elections and remains buffeted by the persistent coronavirus pandemic. The list of...