FDA Approves First-Ever TK2d Therapy

Earlier this week, the U.S. Food and Drug Administration (FDA) approved UCB’s Kygevvi (doxecitine and doxribtimine) to treat the mitochondrial disease thymidine kinase 2 deficiency (TK2d).

Kygevvi is the first FDA-approved therapy for TK2d and marks the second FDA-approved therapy to treat a form of mitochondrial disease this year. Forzinity was approved for Barth syndrome in September of 2025.

“It’s hard to put into words what today’s decision means for patients living with TK2d,” said United Mitochondrial Disease Foundation President and CEO Kristen Clifford. “Too many families have had to endure the terrible burden of this disease. The approval of the first-ever therapy for TK2d is more than a medical milestone – it’s a moment of hope, validation, and possibility for every patient and caregiver who has never stopped believing.”

UMDF has long been a leader in the TK2d community, having hosted an FDA Listening Session focused on the disease in 2022; and just this past June, a Masterclass with a TK2d focus at Mitochondrial Medicine Conference 2025  to help clinicians identify and provide superior care to affected patients.

MELAS Patients & Caregivers, We Need Your Voice for FDA Meeting

On February 10, 2026, UMDF will join MitoAction and MDA in cohosting a groundbreaking Externally-Led Patient Focused Drug Development (PFDD) meeting on MELAS. This is an unprecedented opportunity to bring the voices of patients and caregivers directly to the U.S. Food and Drug Administration (FDA), researchers, and industry leaders who are shaping the future of treatments.

Your experiences, the daily challenges, the impact on your family, and your hopes for better therapies, are the most powerful evidence we can present. The FDA depends on this input to understand what truly matters to our community when it comes to new treatments and clinical trials.

To prepare for this meeting, we need your help. Please take a few minutes to complete our MELAS Patient & Caregiver Survey. Your responses will shape the themes, topics, and priorities that are highlighted at the PFDD meeting. Open Now through November 15!

Click here to take the MELAS Patient & Caregiver Survey.  REMEMBER, the survey must be completed in one sitting – answers are not saved if you leave the window.

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 mitoSHARE today at umdf.org/mitoSHARE. 
 

Florida Energy for Life Event Finishes Out the 2025 Season

Energy for Life walks have already raised hundreds of thousands of dollars to support UMDF’s mission – and there’s still an opportunity to add to that this Saturday, November 8 in Orlando.

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.

Take Action to Support DCA Approval for PDCD Patients

As we shared in August, UMDF has joined with MitoAction, Cure Mito Foundation, Hope for PDCD, and the Elizabeth Watt PDCD Research Fund, to collectively express our deep disappointment with the FDA’s decision not to approve dichloroacetate (DCA) for the treatment of the mitochondrial disease, Pyruvate Dehydrogenase Complex Deficiency (PDCD). While advocacy groups and the manufacturer work behind the scenes to affect change in the situation, you can help support this cause using the below links:

UMDF Advocacy Action: Ask Congress to Encourage "Regulatory Flexibility" for Rare Disease Therapies like DCA

Change.org Petition: FDA: Approve the First Targeted PDCD Treatment and Provide Hope for Our Kids

NATIONAL AUTHOR'S DAY - NOVEMBER 1
KSS Patient and Mom Talk About Self-Publishing Their “Children’s Chapter Adventure” Book

Thomas Gordon is not your typical author. Diagnosed with the mitochondrial disease Kearns-Sayre syndrome in 2014, he lives life totally blind, relies on hearing aids, and cannot walk, hold a pen, or type. But none of that has made a dent in his creativity. 

“He’s always been full of personality and extremely smart,” said his mother, Melissa. With her help – and “some arguing back and forth,” she laughs – Thomas, now 21, wrote Pearl Tides and the Search for The Pahunas, a “children’s chapter adventure book” about friendship and bravery, all set in an underwater world. The self-published book, which is now available on Amazon, took six years to complete.

Choosing the DIY route required a lot of patience. “But we kept going,” she said. The family did hire a few professionals, including an editor and an artist for the book cover, to make it look and sound professional. All-in, they spent a few thousand dollars.

It’s a process they would recommend to anyone in the mito community interested in becoming an author. “Thomas is so proud of himself. And it was a lot of fun.”
 

Have a book suggestion? Continue the conversation on UMDF’s National Author’s Day post on Facebook.