September 2025 Newsletter: World Mito Week, FDA News, Return of Ask The Mito Doc & Bench-to-Bedside

Signup to receive the UMDF newsletter in your email monthly at umdf.org/newsletter-signup.

UMDF Preps for Mitochondrial Disease Awareness Week

UMDF is excited to once again unite with other mitochondrial disease-focused patient advocacy groups across the globe to spread awareness on World Mitochondrial Disease Week, Monday, Sept. 15 to Sunday, Sept. 21. Together, we can promote understanding and recognition with the hope that increased awareness, attention, and funds will move the needle toward effective treatments and cures for the entire mito community.

Below are highlights around World Mitochondrial Disease Week:

• TK2d Tuesday – Tuesday, Sept. 9 Supported by UCB
(CLICK HERE for shareable TK2d graphics)
• UMDF Support Group for All – Wednesday, Sept. 10
• 6th Annual Next Generation Therapeutics Panel hosted by CHOP – Friday, Sept. 12
• Energy for Life Events in Kansas City and Northwest Arkansas – Saturday, Sept. 13
• UMDF Energy for Life National Walk (Virtual) – Sept. 14 thru Sept. 20
• Parent and Caregiver Virtual Support Meeting – Monday, Sept. 15
• UMDF Ask the Mito Doc Monthly Webcast – Tuesday, Sept. 16
• Leigh syndrome Awareness Day – Wednesday, Sept. 17
• LHON Awareness Day – Friday, Sept. 19
• Energy for Life Events in Southern Wisconsin, Western New York – Saturday, Sept. 20
• UMDF Teen & Young Adult Virtual Meet-Up, Ages 16-35 – Friday, Sept. 19
• Light Up for Mito – Saturday, Sept. 20
• Mitochondrial Education Day hosted by CHOP – Monday, Sept. 22

Here’s how you can make a difference:
- Visit our special WMDW social media content page where you can download custom images.

- Watch UMDF social media for opportunities to participate in events, share content, and more. Share your #mitostory and tag us @umdf all week long. Use the hashtags #WorldMitoDiseaseWeek2025, #DecodetheMitoPuzzle, #LightUpforMito and #HopeWithinReach.

- Make requests for local buildings/monuments to light up GREEN on Sept. 20.

September Ask the Mito Doc Focuses on Seizures

Join us on Tuesday, Sept. 16 at 7pm ET for an Ask the Mito Doc webinar focused on Seizure Management in Patients with Mitochondrial Disease featuring Russell Saneto, DO, PhD, of Seattle Children’s Hospital.

Click here to register. / Click here to submit your questions in advance.

UMDF Bench-to-Bedside Seminar Looks at Rehab Medicine

Researchers and clinicians, join us for our upcoming Bench-to-Bedside Seminar focused on The Role of Rehabilitation Medicine in the Care of Children on Sept. 8 at 12pm ET. Click here to register.

Speakers/Panelists include:

Dr. Afua Asante-Otoo, MD, FAAPMR, National Institutes of Health, Staff Clinician / Pediatric Physiatrist, Rehabilitation Medicine Department

Hanna Hildenbrand, MS, OTR/L, National Institutes of Health, Occupational Therapist, Rehabilitation Medicine Department

Dr. Minal Jain, PT, DSc, FAPTA, National Institutes of Health, Acting Chief Physical Therapy Section, Rehabilitation Medicine Department

Beth Solomon, MS, CCC-SLP, National Institutes of Health, Senior Lead Speech-Language Pathologist, Rehabilitation Medicine Department

FDA Declines to Approve PDCD-Focused Therapy

Yesterday, UMDF shared the FDA has denied approval of Saol Therapeutics' dichloroacetate (DCA) to treat Pyruvate Dehydrogenase Complex Deficiency (PDCD).

UMDF joined with fellow patient advocacy groups MitoAction, Cure Mito Foundation, Hope for PDCD, and the Elizabeth Watt PDCD Research Fund to express "deep disappointment" with the decision. You can read the full statement on our umdf.org blog.

Click here to read a community statement from Saol. The company plans to host a community townhall on Thursday, Sept. 18 at 3pm ET. Watch for an invite in the coming days.

We encourage you to reach out to your members of Congress to help them better understand PDCD and the importance of regulatory flexibility for therapies like DCA. Click HERE for a form letter that will be sent straight to your representative.

MELAS Patients Needed for Clinical Trials, Adolescent Study, Registry

Calling all in the mito community with MELAS (mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes) -- there are several studies and clinical trials open right now for people diagnosed with MELAS, including clinical trials with Tisento and Abliva/Pharming! Visit umdf.org/clinical-trials or reach out to mailto:registry@umdf.org with questions about eligibility.

Also open is Tisento’s Adolescents with MELAS Interview Study, open to English-speaking MELAS patients ages 12 – 17. Contact MELAS@adelphivalues.com or call (617) 720-0001 if interested.

And as always, make sure to join mitoSHARE to keep up with the latest surveys and studies – including a soon to be launched MELAS study! Join today mitoSHARE at umdf.org/mitoSHARE.

Join an Upcoming Energy for Life Walk
There are still numerous opportunities to help energize the fight at an upcoming Energy for Life walk this year!

Energy for Life events help fund mitochondrial disease research, education, and advocacy, while supporting and connecting the mito community. Last year, such walks brought in over $500,000 from across the country, money that is crucial in helping chart the course toward mitochondrial disease treatments and eventual cures.

If you or someone you know is interested in doing more for the mito community, contact us to learn how you can be a part of a planning committee for an Energy for Life event! Get involved by visiting energyforlifewalk.org.

Saturday, Sept. 6, 2025	Indianapolis
Sunday, Sept. 7, 2025	Chicago
Sunday, Sept. 7, 2025	Delaware Valley
Saturday, Sept. 13, 2025	Kansas City
Saturday, Sept. 13, 2025	Northwest Arkansas
Sept. 14-20, 2025	National Walk (Virtual)
Saturday, Sept. 20, 2025	Southern Wisconsin
Saturday, Sept. 20, 2025	Western New York
Saturday, Oct. 4, 2025	Charlotte
Sunday, Oct.5, 2025	Seattle (Virtual)
Saturday, November 8, 2025	Orlando

	MitoWorld’s #BeyondTheDisease 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 Annual Virtual Leigh Syndrome Symposium on Tuesday, Sept. 16, 2025 – plus, Mitochondria in Space, How Evolution Shapes the OxPhos Machinery, Mitochondrial DNA Mutations and Cancer, Old Mitochondrial Are a Factor in Cell Fate Decisions, and Turning Off Translation of Mitochondrial Genes. Click here to read the blog.
	Mito Med Co-Chairs Announced for 2026 Carlos Moraes, PhD of Miller School of Medicine at the University of Miami, and Fernando Scaglia, MD of the Baylor College of Medicine, have been named co-chairs of upcoming Mitochondrial Medicine Conference on June 17-20, 2026 at the Renaissance Orlando at SeaWorld. Registration is expected to open in January. Stay tuned to umdfconference.org and UMDF social media for more details.
	Patient-Focused MitoMed 2025 Videos Now Available for Viewing Videos of all patient-focused sessions from Mitochondrial Medicine 2025 in St. Louis this past year are now available on UMDF’s YouTube channel. Amongst the topics available are sessions on mental health, palliative care, shared wisdom, ICU visits, Ask the Mito Doc, and much more.
	FBXL4 & PPTC7 Post-Doc Research Project Funded Last week, UMDF announced funding for Keri-Lyn Kozul, PhD for her project, "Therapeutic degrader molecules targeting excessive mitophagy in FBXL4- and PPTC7- associated Mitochondrial Disease." Keri-Lyn was featured earlier this year as a finalist in UMDF's accelerators program. Funding support was generously provided by The Kamaria Satcher Fund for Kearn’s-Sayre Syndrome at UMDF. FBXL4 and KSS are both examples of mitochondrial deletion and depletion syndromes. Learn more about UMDF’s commitment to funding mitochondrial research at umdf.org/research.
	Elamipretide Review Takes Positive Turn Following extensive advocacy from the Barth Syndrome Foundation and mito communities, Stealth BioTherapeutics announced the FDA has accepted for review the resubmission of the New Drug Application for elamipretide for the treatment of Barth syndrome. While the FDA considers the resubmission a complete, class 2 response with a six-month timeline, the agency has advised that its planned goal date is Sept. 26, 2025.
	Vatiquinone Denied Approval for Friedreich's Ataxia The FDA has denied the New Drug Application for PTC Therapeutics’ vatiquinone for the treatment of children and adults living with Friedreich's ataxia. PTC has shared that while they work with FDA to understand the next steps for vatiquinone, they will continue all ongoing vatiquinone studies and continue to provide vatiquinone for all those participating in the studies for the foreseeable future. If you’re a PMD patient currently on the therapy as part of IND (investigational new drug application) and have questions, we suggest you reach out to your clinician. We’ll do our best to keep the community informed.