At 19, Walker Burger had already lived through years of unexplained exhaustion, muscle weakness, and mysterious symptoms that defied easy classification. He looked like a typical young adult in Monroe, Georgia—but something was always different. And he knew it.

That’s when he finally got the answer: Barth syndrome. A rare and potentially fatal mitochondrial disease that affects fewer than 200 individuals in the U.S., Barth syndrome often goes undiagnosed for years—sometimes with devastating consequences.

Walker was one of the lucky ones—not just for finally getting a diagnosis, but for what came next.

In 2017, Walker enrolled in the clinical trial for elamipretide, a first-in-class investigational drug designed to improve mitochondrial function and energy metabolism. “It changed my life completely,” he says now. “I’ve been on it for over seven years, and it’s the reason I’m living as fully as I am today.”

The drug, developed by Stealth BioTherapeutics, is currently under review by the U.S. Food and Drug Administration. But in a move that has alarmed patients and physicians alike, the FDA recently delayed its decision on whether to approve it—even after an advisory committee voted 10–6 in favor of its effectiveness for Barth syndrome.

For Walker, the delay is more than policy. It’s personal.
“I just want to be able to continue taking this medication,” he says. “I worry that I will lose access to the drug that has changed my life. But more importantly, I want others to have access to it too. Everyone living with this disease deserves the chance I had. I hope the drug is approved for all ages—especially for those who have been put on it for emergency use, including the infants, as they need it the most.”

Like many with Barth syndrome, Walker’s symptoms were subtle yet debilitating: low stamina, vulnerability to infections, skeletal muscle weakness, and the ever-present fatigue that made simple tasks feel insurmountable. But unlike others, he got the chance to see what life could be like with an effective treatment. The impact wasn’t abstract. “The opportunity to stay on the drug would change my life forever.”

Before elamipretide, his life was a careful calculation of energy and risk. On it, Walker found new strength and stability. “I could do more,” he says simply. “I wasn’t just existing—I was finally living. Because of the drug, I’m able to live the life I could only dream about before.”

Today, at 36, Walker says he “lives a life without the symptoms of Barth”—something that would have been unthinkable a decade ago. He keeps up with family. He participates in his community. He works. But all of that could unravel if access to the drug is pulled—or if others are denied it altogether.

That’s why he’s speaking out.

As someone who participated in the TAZPOWER trial and continues on the therapy through compassionate use, Walker sees himself not just as a patient but as a living testament to what’s possible when science and policy align.

The Barth Syndrome Foundation and a coalition of rare disease advocates have issued a public call urging the FDA to complete its review and approve the therapy for all individuals with Barth syndrome. They’re asking for action—not more waiting.

For Walker, it’s about making sure the next 19-year-old doesn’t have to wait as long—or suffer as much—for the same chance.
“This is bigger than me,” he says. “I’ve had the privilege of access. Now it’s time to make sure others do too. I hope one day all boys and their families living with Barth syndrome can live the life I’m living now… because it’s truly the life I could only dream about before.”

Call to Action
The Barth Syndrome Foundation is urging the FDA to:
• Finalize its review of elamipretide without further delay
• Set a definitive decision date
• Approve the drug for the full population of individuals with Barth syndrome

To support families like Walker's, visit www.barthsyndrome.org or read the foundation’s full statement: Barth Syndrome Foundation Calls for Urgent FDA Action