February 2026 Newsletter: Rare Disease Awareness, Advocacy Updates, and More

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February is National Heart Month and Rare Disease Month

For the mitochondrial disease community, heart health is part of everyday health. Join us throughout the month as we highlight the important connection between mitochondrial disease and heart health. Have a mito heart story to share? Tag us on social media @UMDF #MyMitoHeart.

In honor of February also being Rare Disease Month, our CEO, alongside UMDF staff members, and members of the mito community, will take part in congressional meetings on Capitol Hill ahead of Rare Disease Day on Saturday, February 28. Keep an eye on our social media channels for live updates, as we continue to advocate for the voices in the mitochondrial disease community.

Get a Glimpse of the MitoMed Agenda

You can now get a sneak peek at programming for Mitochondrial Medicine Conference 2026 in Orlando this summer by visiting umdfconference.org. Hear from the leading names in medicine and science, including special disease-focused programming for Leigh syndrome families, and MELAS-focused programming for the clinical and scientific community. Registration and scholarships will open later this month.

Click here to book your room.

Ask Your Representative and Senators to Cosponsor the 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. You can take action now on the UMDF Advocacy Center to ask your local members of Congress to join as co-sponsors: Cosponsor Medical Foods and Formulas Access Act

The First-Ever PPA2 Virtual Conference is Coming Up

The first-ever PPA2 Virtual Conference is right around the corner on Friday, February 13 at 12PM EST via Zoom! UMDF is honored to join the medical community to bring you this conference organized and moderated by Amy Goldstein, MD and Hilary Vernon, MD, PhD., focusing on “Connecting Clinical Care and Scientific Discovery.” While the conference is open to all members of the mitochondrial disease community, the topics are geared toward researchers and clinicians. Click here to register.

Calling All Mito Researchers

UMDF, in partnership with The Mito Foundation, is pleased to announce that the 2026 Research Grant Program is now open.

The 2026 Research Grant Program, co-funded by UMDF and The Mito Foundation, emphasizes advancing disease-focused research across basic, translational, and clinical domains, and training and workforce development for the next generation of mitochondrial disease researchers. Available grants include the Graduate Student Award, Postdoctoral Fellowship Award, and Principal Investigator Award.

Proposals must be submitted by Friday, February 27, 2026. Click here to apply.

Save the Date for the Next Bench-to-Bedside Webinar

During this month’s Bench-to-Bedside seminar on Monday, February 9 at 12pm EST, join us for a MitoCon 2026 Debrief: Emerging Trends, AI Tools, and Translational Progress. We’ll be hearing from Michelangelo Mancuso, MD, PhD, from the Neurological Institute at the University of Pisa & AOUP in Italy. To register, click here.

If you missed the last Bench-to-Bedside webinars, you can watch the replays on Mitochondrial Disease Clinical Trial Updates Part I and Part 2.

UMDF to Brief Lawmakers on Mito Therapy Development

UMDF will be on Capitol Hill rare disease week for a Mitochondrial Disease Caucus Briefing on “From Discovery to Delivery: What the FDA Drug Review Process Means for Patients and Providers.” Joining UMDF President & CEO Kristen Clifford will be Hope for PDCD Founder and CEO Frances Pimentel and several affected families who will discuss their experience with Pyruvate dehydrogenase complex deficiency and the urgent, unmet medical need for an approved therapy.

The Mito Doc Answers Your Questions

In our latest Ask the Mito Doc Webinar on Tuesday, January 20, we discussed Cardiac Mito with clinicians and offered genetics and clinical insights to jumpstart the year. We were joined by Hilary Vernon, MD, PhD, from Johns Hopkins University School of Medicine, Andreas S. Barth, MD, PhD, FAHA, from Johns Hopkins University, and Rebecca McClellan, MGC, CGC, from the Kennedy Krieger Institute. Catch the replay here.

Join us for the next Ask the Mito Doc Webinar on Tuesday, February 17 at 12pm EST on Understanding MELAS: Consensus Updates, Patient Perspectives, and Community Support with Amel Karaa, MD, from Massachusetts General Hospital.

Click here to register and submit your question.

Run for Mitochondrial Disease Research in the 2026 Bank of America Chicago Marathon

“A truly life-changing experience”. That’s what Diego Santos, who ran the 2025 Bank of America Chicago Marathon last year as part of UMDF’s Team Activate, called his run.

This year, join a group that’s determined to raise awareness and funding for mitochondrial disease research and the families that depend on it.

Diego credited UMDF with supporting his training and fundraising efforts and encourages you to participate: “Just do it! It’s an amazing opportunity and an even greater cause.”

If you’re interested in being a part of UMDF’s Team Activate and running the 2026 Bank of America Chicago Marathon on October 11, complete our prescreening form to get started.

Become a singlet sponsor
Feature your company logo on the back of Team Activate singlets at the 2026 Bank of America Chicago Marathon to support the UMDF mission. Learn more by contacting tara.gallessich@umdf.org.

We’re a Proud Member of REN

UMDF is proud to share we have joined The Rare Epilepsy Network (REN), a collection of members and partner groups focused on improving the outcomes of people with rare epilepsies. Research has estimated as many as one in every four mito patients experience some form of epilepsy. Click here to learn more about REN.

Do You Have Primary CoQ10 Deficiency? If you have Primary CoQ10 Deficiency, we want to hear from you! Your insights could support upcoming research and regulatory efforts. Click here to join mitoSHARE today.

Join Energy for Life Walks as a Sponsor or Volunteer UMDF’s Energy for Life Walks kick off this spring! Support mitochondrial disease research and the families who depend on it by becoming a corporate sponsor or committee volunteer. To learn more, email Tara Gallessich at tara.gallessich@umdf.org to become a corporate sponsor or Kelsi Martinez at Kelsi.martinez@umdf.org to become a committee member.

Join the Externally Led Patient-Focused Drug Development Meeting on MELAS In collaboration with MitoAction, International Mito Patients, and the Muscular Dystrophy Association, UMDF is proud to co-host a virtual Externally-Led Patient-Focused Drug Development (PFDD) meeting on MELAS (Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like Episodes) on February 10 from 10am-4:30pm EST. This webinar is open to all members of the mitochondrial disease community, including patients, caregivers, clinicians, researchers, and industry partners. Click here to register.

Saol Therapeutics Provides FDA Updates
Saol Therapeutics has provided updates on a potential dichloroacetate (DCA) resubmission to the US Food and Drug Administration (FDA). In a letter addressed to the PDCD community, Saol CEO Dave Penake said the company had provided the FDA “additional analyses describing functional benefit, new survival analyses, mechanistic evidence that SL1009 (DCA) addresses the underlying enzyme deficiency in PDCD” – enough so that the FDA has planned a follow-up meeting this spring. Penake added that the company is “encouraged by this progress and remain hopeful that this represents a meaningful path forward for the program.” You can read the full letter here.

MitoWorld Each month, UMDF partners with MitoWorld for #BeyondTheDisease, a feature highlighting advances in mitochondrial science and the people responsible for them. This month, read about the upcoming FUSION Conference, MitoWorld’s new partnership with Heureka Labs, and a recent paper in Nature Metabolism. Click here to read more.