Advocacy Center
Congress needs to hear your voice! The UMDF Advocacy Center is your one-stop page for supporting legislation that helps move mitochondrial disease research, education and support forward.
Read our latest news, sign-up for future congressional visits, or take action today via our UMDF Action Center.
UMDF ADVOCACY ITEM
Ask Congress to “encourage regulatory flexibility” for Rare Disease Therapies like DCA for PDCD
Saol Therapeutics has shared the U.S. Food and Drug Administration (FDA) will not approve Saol Therapeutic’s New Drug Application for SL1009 (DCA) for Pyruvate Dehydrogenase Complex Deficiency (PDCD) in its current form, outlining specific observations that must be addressed before potential approval can be reconsidered.
While we fully support rigorous scientific standards, regulatory flexibility is essential in the rare disease community and we are asking the Congressional offices to reach out to senior officials at the Department of Health and Human Services (HHS) to discuss the value and importance of regulatory flexibility in the rare disease space.
UMDF ADVOCACY ITEM
Ask For Your House Rep & Senators to Cosponsor Medical Foods and Formulas Access Act
The Medical Foods and Formulas Access Act, which would require coverage of “medically necessary food” for individuals who receive health coverage through Medicaid, CHIP, Medicare and FEHBP, has been introduced in the U.S. House of Representatives and the Senate and is looking for cosponsors. A recent medical nutrition survey of UMDF families found that nearly 25% were spending $250 or more out of pocket monthly on such items. Ask your local members of Congress to join as co-sponsors now using the below link.
UMDF REGULATORY ITEM
FDA Asks for Comments on PFDD Meetings
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The U.S. Food & Drug Administration has opened a public docket seeking real-world examples of how Patient-Focused Drug Development meetings — like externally led PFDDs and listening sessions — have influenced drug development, research priorities, advocacy strategies, and clinical practice. It is UMDF’s stance that for conditions as complex, diverse, and historically neglected as mitochondrial disease, the PFDD process keeps drug development anchored to the lived reality of patients and families. Contribute your voice by submitting comments by 7/31/26. (NOTE: All comments are public) |
UMDF REGULATORY ITEM
FDA Asks for Comments on Petition to Revise Rare Disease Drug Development
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The U.S. Food & Drug Administration has opened comments on a petition from Haystack Project, Inc. The petition suggests two regulatory changes, including:
UMDF is supportive of the petition and encourages those in the mitochondrial disease community to add their voice via Regulations.gov. (NOTE: All comments are public) |
of mitochondrial disease research from the U.S. Department of Defense
from National Institutes of Health
members of the Mitochondrial Disease Caucus in the U.S. House of Representatives
total UMDF Advocates pushing for mito-friendly legislation
Saol Therapeutics Provides Update from Type C Meeting with FDA regarding DCA
UMDF Co-Signs Letter to CMS Laying Out How Proposed ACA Changes Could Affect Existing Medical Nutrition Coverage
UMDF, LHON Collective Issue Statement on the FDA’s Decision Not to Approve Idebenone
UMDF is disappointed by the U.S. Food and Drug Administration’s (FDA) decision not to approve idebenone for Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease that causes sudden and often irreversible vision loss. “Far too many patients continue to...
UMDF Cosigns Letter to Congressional Leaders Urging Passage of Give Kids a Chance Act and Accelerating Kids’ Access to Care Act
UMDF Joins Fellow Mito PAGs on Letter to Senate Special Committee Regarding FDA Flexibility for DCA
READ THE LETTER: Leading Clinicians and Researchers Urge FDA to Reconsider Path Forward for DCA in Pyruvate Dehydrogenase Complex Deficiency
Click the Expand Button in the bottom right on the widget for a larger, full-screen view of the PDF.
UMDF Co-signs Letter Encouraging Congress to “Act Swiftly” to Reauthorize the Rare Pediatric Disease PRV Program
UMDF 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 Letter (CRL) regarding the company’s application for dichloroacetate (DCA) to treat Pyruvate Dehydrogenase Complex Deficiency...
Advocacy Action: Ask FDA to Reconsider Elamipretide Decision
Barth Syndrome Foundation, UMDF, and MitoAction leadership were recently notified by Stealth BioTherapeutics that FDA has refused their request to reconsider the elamipretide new drug application (NDA). This refusal to reconsider comes despite a positive Type A...
UMDF Cosigns Letter to HHS Urging Preservation of Federal Newborn Screening System
Ask Your House Representative to Support Mito Research Funding in 2026 Appropriations
UMDF Cosigns Letter to Congressional Leaders Supporting Accelerating Kids’ Access to Care Act
Advocacy Action: Ask Congress to Oppose 57% Cut to Congressionally Directed Medical Research Programs
Updated 3/12/25, 11amLast night, the House approved the continuing resolution -- including the 57% CDMRP cut -- to fund the government through September 30 with a final vote of 217-213. The measure still needs to pass the Senate, however, where it will need bipartisan...
UMDF Cosigns Letter to New Administration Outlining Rare Disease Priorities
UMDF Signs On to Letter to Congress Supporting Accelerating Kids’ Access to Care Act
UMDF November Advocacy Update: What Does a Second Trump Administration Mean for the Mito Community?
With the recent election results, we’ve been getting some inquiries about the impact of a second Trump Administration on mitochondrial disease patients and families. And the truth is, it’s too early to know – but our governmental relations team at Holland & Knight...
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
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
Advocacy Update – UMDF Co-Signs Letter Supporting PRV Reauthorization
UMDF Co-Signs Letter Supporting Rare Pediatric Disease Priority Review Voucher Reauthorization
Advocacy Update – UMDF Signs On to Thank You Letter to Senate Appropriations Leadership for Proposed NIH Budget Increase
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...
UMDF Issues Response to Congressional 21st Century Cures Act RFI
UMDF Issues Response to Congressional 21st Century Cures Act RFIDOWNLOAD this document (PDF) >>
Advocacy Invite: FDA to Host Patient and Care Partner Perspectives on Safety Considerations for Approved Gene Therapy Treatments for Rare Diseases on Friday, September 20
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...
Advocacy Update – July 2024: Watch Mito Caucus Briefing, A Win with NIH Language, and more
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,...
Advocacy Update – May 2024: FAA Reauthorization, Telehealth, Accelerating Kids’ Access to Care Act, and more
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...
Advocacy Update – February 2024: Redefining “Rare” For the World, Funding for 2024 & Beyond
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...
Advocacy Update – January 2024: New FDA Guidance Encouraging Sign for Rare Disease Patients
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...
Advocacy Update – December 2023
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...
Advocacy Update – November 2023: CMS Changes Definition of Medicare Caregiver in New Rule
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...
Language Encouraging NIH to “Expand, Accelerate, and Collaborate” on Mito Research Makes it in HHS Appropriations Report
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...
Marking #WorldMitoWeek Through Advocacy
(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 Reacts to Today’s Historic FDA Listening for the Mitochondrial Disease PDCD
(Sept. 8, 2023) Earlier today, the United Mitochondrial Disease Foundation (UMDF) cohosted a listening session with the U.S. Food and Drug Administration (FDA) with fellow advocacy group MitoAction regarding the mitochondrial disease Pyruvate Dehydrogenase Complex...
Advocacy Update – May 2023: What Does the End of the Public Health Emergency Mean for Medicaid?
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....
Advocacy Update – April 2023: Spring on Capitol Hill
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...
Advocacy Update – March 2023: Federal Appropriations 101 and Why Advocacy Matters for Mito
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...
Advocacy Update – February 2023: Could ARPA-H Change the Research Landscape for Rare Diseases?
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...
Advocacy Update – January 2023: What Does 2023 Hold for Mito Advocacy?
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...
Advocacy Update – December 2022: Mito Research Funding Looks Headed for Renewal in Newly Unveiled Funding Bill
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...
Advocacy Update – September 2022: Three Things You Should Ask of Congress as We Wrap Up World Mitochondrial Disease Week
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...
UMDF Marks World Mitochondrial Disease Week, September 18 – 24, 2022
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...
Advocacy Update – August 2022: The Inflation Reduction Act, Telehealth Extension, FY 2023 Appropriations and more.
Congress Passes the Inflation Reduction Act Congress ended a busy work period after passing the Inflation Reduction Act (IRA) over the weekend. Congress has now adjourned for August recess. The Senate returns on September 6, and the House returns on September...
Advocacy Update – May 2022: CDER Launches Program to Speed Development of Rare Disease Treatments
The Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER) announced the launch of the Accelerating Rare disease Cures (ARC) Program. CDER’s Rare Diseases Team manages the ARC Program. The ARC program’s mission is to speed up the...
Advocacy Update – April 2022
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...
Advocacy Update – March 2022
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,...
UMDF’s 2022 Congressional Outlook
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...
UMDF Updates Congress on Telehealth
Before the pandemic, telehealth coverage was limited in scope. However, the Centers for Medicare & Medicaid Services (CMS) and Congress expanded coverage to encourage broad adoption of telehealth for the duration of the public health emergency. Accordingly, we have seen an exponential increase in telehealth.
Powerhouse Podcast Episode #1 – A Conversation Adult Patient Brandon Leach
Powerhouse Podcast Episode #1 UMDF President and CEO Brian Harman and Science and Alliance Officer Dr. Phil Yeske debut the Powerhouse Podcast with a conversation with Brandon Leach. Brandon is a resilient young adult patient navigating mitochondrial disease, college...