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In early October, 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 males affecting the heart, immune system, and muscles, amongst other things.

“The committee’s decision reinforces our belief that, when it comes to families experiencing mitochondrial disease, patients deserve access to any therapy proven to safely improve quality of life for these devastating diseases,” said UMDF’s Interim Managing Director and Science and Alliance Officer Dr. Philip Yeske, who was on-hand for the historic vote.

He also praised the work of the Barth Syndrome Foundation and the many families and clinicians who testified.

“The open public hearing portion of the meeting was extremely powerful and seemingly played a critical role in turning the tide toward a recommendation for approval. I think that’s a reflection of the closeness of this community, and the hard work of the Barth Syndrome Foundation.”

The advisory committee's recommendation helps guide the FDA’s decision, with a final determination on elamipretide expected by January 29, 2025.

See the March Toward Approval on UMDF’s Mitochondrial Disease Therapeutic Pipeline

For the latest news on trials and therapies, visit UMDF’s Mitochondrial Disease Therapeutic Pipeline page, which is regularly updated. In October, we added a new trial and updated the recruiting statuses of several others. All studies are organized by mitochondrial disease type with a bar graph showing progress. Learn more at Therapeutic Pipeline | UMDF.

In the News
Rare Revolution Magazine Features UMDF Leader, Volunteer in October Issue

Rare Revolution Magazine featured several UMDF leaders as part of a "RARE mitochondria" issue in October.

In an article titled "The Science of Mitochondria -- Unlocking the Full Potential through Collaborative Research," UMDF Interim Managing Director and Science & Alliance Officer Dr. Philip Yeske joined Dr. Michio Hirano of Columbia University and team members from the U.K.-based patient advocacy group, The Lily Foundation, to talk about the need for collaboration in the mito space.

In a separate piece, the magazine honored UMDF Support Ambassador and long-time volunteer, Linda Ramsey. Linda has volunteered for over two decades raising mitochondrial disease awareness, providing support for mito patients and families, and fundraising toward research.

Spots Now Open on UMDF's "Team Activate" for 2025 Bank of America Chicago Marathon

Gain access to one of the most exclusive marathons in the country while supporting the mitochondrial disease community.

We're now recruiting runners for the 2025 Bank of America Chicago Marathon as a part of UMDF's "Team Activate."

Runners on UMDF’s team are provided guaranteed entry to the Bank of America Chicago Marathon on October 12, 2025, with no lottery or other qualifying requirements to run. Participants do have a fundraising commitment in return for the guaranteed entry and are responsible for their registration fee and associated travel costs.

We encourage you to act now, as spots are limited. For full details, contact UMDF Development Coordinator Angela Sommer at angela.sommer@umdf.org or 412.539.9450.

Adolescent Patients with MELAS Needed for Study

Adolescents living with the mitochondrial disease MELAS are needed to collect perspectives that will help shape how the effects of a drug in development will be assessed in an upcoming clinical trial.

The study is open to any English-speaking patients ages 12 – 17 who are clinically and genetically diagnosed with MELAS. Participants will receive $150 for completing two 45-minute interviews ($75 per interview) from the comfort of your home and at your convenience.

Click here for more information and details on how to enroll.

Related Posts:
UMDF Surveys & Studies page

Energy for Life 2025 Planning Underway

As we celebrate another slate of successful Energy for Life events -- nearly $500,000 was raised to fund vital UMDF mission programs! – UMDF volunteer leaders from across the country are already making plans for 2025. There are numerous ways to get involved now, ranging from walking, making plans for a team, or becoming a team captain. Get involved now by visiting energyforlifewalk.org or emailing events@umdf.org.

Save the Date for #GivingTuesday Next Month

UMDF will once again participate in Giving Tuesday, a global generosity movement unleashing the power of people and organizations to transform their communities and the world.

This year, Giving Tuesday falls on Tuesday, December 3, 2024. Click here to make your gift – and remember to check if your employer offers a match!

Help Researchers Understand the Use of Assistive Technology in the Mito Community

Your voice is needed for a new mitoSHARE study focused on the use of assistive technology within the mitochondrial disease community.

The Assistive Technology Survey, which will launch in the coming weeks, assesses the needs of mito patients to help better understand what assistive technology products you use, what products you would like to use but don't, and why. This is a short survey that should take less than five minutes to complete. You can lend your voice to this by joining mitoSHARE today at umdf.org/mitoSHARE.

mitoSHARE, UMDF’s patient registry, is a worldwide research database for patients (both living and deceased) with any form of mitochondrial disease, as well as their caregivers. A confirmed diagnosis of mitochondrial disease is not required to join mitoSHARE. Patients at any stage of the diagnostic journey are encouraged to register.

Gold Medal Paralympian & Mito Patient Olivia Chambers Joins UMDF’s Powerhouse Podcast

Watch October’s Powerhouse Podcast as UMDF’s Cassie Franklin talks with Olivia Chambers, a college athlete at the University of Northern Iowa and a 2024 Paralympic gold and silver medalist. Olivia talks about her passion for distance swimming, being a Paralympian, her mito story and life with vision loss. Find it wherever you source your podcasts or watch via the link below.

WATCH: October Powerhouse Podcast featuring Paralympic Medalist Olivia Chambers

For Clinicians & Researchers
Bench-to-Bedside

Join us for the next Bench-to-Bedside webcast on Monday, November 11 at 12pm ET focused on Current Diagnostic Strategies and Challenges in the Diagnosis of Mitochondrial Disease in Argentina. The sessions moderators are Fernando Scaglia, MD and Zuela Zolkipli Cunningham, MD, who are joined by speakers Mariana Loos, MD, of Hospital de Pediatría "Juan P. Garrahan," Argentina, and Soledad Kleppe, MD, of Hospital Italiano de Buenos Aires, Argentina. Click here to register.

WATCH: Bench-to-Bedside – October 2024: Novel Approaches for Achieving a Molecular Diagnosis in Mitochondrial Disease Patients | View full archive of Bench-to-Bedside Seminars.

For Families
Ask the Mito Doc

Join us for an Ask the Mito Doc webcast on Wednesday, November 20 at 7pm ET focused on Understanding POLG Disorders. Speakers include William Copeland, PhD, National Institute of Environmental Health Sciences (NIEHS), of Research Triangle Park and Bruce Cohen, MD, of Akron Children’s Hospital. Click here to submit questions ahead of time. Click here to register.

WATCH: Ask the Mito Doc - October 2024: Connecting the Dots Between Mitochondrial Disease & Rare Diseases | View full archive of Ask the Mito Doc Webinars.

Highlighted Support Meetings and Events

Prep for Clinical Trials with UMDF’s Newest Group

Clinical trials are our greatest hope for finding treatments and cures for mitochondrial disease. Are you ready? Join our new Clinical Trial Readiness and Information Group, which will meet monthly starting Tuesday, November 19 at 12pm ET for a talk on clinical trial basics. Click here to register.

November 7, 8pm ET -- Adult Virtual Support Meeting

November 18, 8pm ET - Parent and Caregiver Virtual Support Meeting

November 19, 12pm ET - Clinical Trial Readiness and Information Group

November 19, 12pm ET - Mindfulness with Mary Meditation Support Series

December 5, 8pm ET - Adult Virtual Support Meeting

View more at support events umdf.org/events-calendar.