MDA 2025: Donavon Decker honored for decades of advocacy
Man with LGMD has taken part in advisory boards, early gene therapy trial
Patient advocate Donavon Decker has been given the 2025 MDA Legacy Award for Community Impact in Research, recognizing his efforts across decades on behalf of the muscular dystrophy community.
Decker, who has limb-girdle muscular dystrophy (LGMD) type 2D, was honored at the Muscular Dystrophy Association (MDA)’s annual meeting, recently held in Dallas and virtually.
“It’s always nice to be recognized for the work that you’ve done over the years,” Decker said in an onsite interview with Muscular Dystrophy News Today.
Among his advocacy work, Decker has served on the LGMD Community Advisory Board and participated in the Muscular Dystrophy Coordinating Committee at the National Institutes of Health (NIH). He is the co-founder of Angle Therapeutics, a company working to develop nonviral gene therapies for all types of LGMD and another undisclosed condition.
‘I’m gonna fight like crazy to help myself, my family, others’
He also is the subject of the short documentary “In Search of Strength,” which was presented at the MDA conference and is available online.
Decker comes from a family of eight siblings, five of whom were diagnosed with LGMD, and two of his nieces also live with LGMD. He said that his family has been the “main driver” of his advocacy work in support of patients, disease research, and in “trying to make a difference.”
Donavon Decker was honored with the 2025 MDA Legacy Award for Community Impact in Research.
Looking ahead, “I’m gonna proceed, and I’m gonna fight like crazy to help myself, my family, and others,” he said in the documentary.
Decker, now 62, was diagnosed with LGMD in 1978 when he was 15 years old. Although many people with this rare disease have to undergo long and arduous journeys to arrive at a correct disease diagnosis, Decker and his siblings were fortunate in having a doctor who quickly recognized the symptoms of LGMD.
“We had a great neurologist … as soon as he basically [saw] our family, he knew what it was,” Decker said.
There are many different types of LGMD, as the disease is caused by a variety of genetic mutations. While Decker knew he had LGMD from the time he was a teenager, the reasons were not understood. It wasn’t until 20 years later that researchers discovered some of the specific mutations that cause LGMD. Decker recalled reading about the discovery in Quest, the MDA’s magazine. This prompted him to undergo testing that revealed he carried mutations in the SGCA gene, allowing him to be specifically diagnosed with LGMD type 2D.
Decker was first MD patient given a gene therapy in an early clinical trial
Around the time that Decker learned the genetic cause of his disease, researchers were beginning to explore the idea of gene therapy, which delivers healthy versions of mutated genes as a way to treat disease. In September 1999, he became the first person with any type of muscular dystrophy to receive an experimental gene therapy as part of a clinical trial.
In the trial, which mainly evaluated treatment safety, an experimental gene therapy was injected into a small muscle in Decker’s foot. He said his doctor, Jerry Mendell, MD, made it clear that a safety study offered virtually no chance of the gene therapy improving his health, while there were risks in the procedure’s unknowns. Still, Decker said he had confidence in his clinical care team as well as the MDA, which helped to back the study.
“If [the MDA] didn’t believe in it, they wouldn’t be risking to do it,” he said.
The trial ended up being terminated early, following the death of a patient in a different trial evaluating a gene therapy for a metabolic disorder called ornithine transcarbamoylase deficiency. That death sent shockwaves through the research community, sparking an onslaught of regulatory concern and effectively cratering gene therapy development for the better part of a decade.
Looking back, Decker said he wishes he had done more to advocate for continuing LGMD gene therapy research, given that the death was related to a completely different gene therapy for a totally different disease. He sometimes wonders if, had he been more vocal, his sisters who died of LGMD complications might have received treatment that could have prolonged their lives.
“That’s something at times [that] really gets to me. Other times it’s like, you can’t change what happened or how things happened,” he said.
Being realistic about gene therapies, while working toward better ones
The gene therapy field slowly recovered over the last decade or two — and gene therapy is now a reality for some in the muscular dystrophy community. Elevidys (delandistrogene moxeparvovec-rokl), a gene therapy developed by Sarepta Therapeutics, was fully approved last year by the U.S. Food and Drug Administration to treat people with Duchenne muscular dystrophy (DMD) ages 4 and older who are able to walk. Elevidys also is conditionally authorized for patients 4 and older who cannot walk.
No gene therapy is approved for LGMD type 2D, although Sarepta has been developing SRP-9004 for this disease form and has conducted early trials. Several gene therapies are in development for other types of LGMD.
Decker noted that, although gene therapy holds immense promise, it’s unlikely to offer a cure and not everyone will benefit from such treatment. He stressed that people looking to undergo gene therapy need to have realistic expectations about what it can and cannot do.
Most gene therapies use a modified virus to deliver a healthy gene to the body’s cells. A major limitation of this approach is that, after treatment, the body usually develops immunity against the virus used. This has been the case for Decker, who still showed signs of immunity against his gene therapy’s viral vector more than a decade later. A delivery system that allows for redosing, Decker said, will be important for a gene therapy’s clinical use, though such strategies are presently in early experimental stages.
Despite not being eligible for subsequent gene therapy dosing, Decker stressed that he has no regrets about his participation in the early trial. He encourages people living with LGMD to get involved in research in any way they can — especially now that many trials have moved beyond preliminary safety studies and are looking into if and how a gene therapy benefits patients.
“You won’t find a person that’s more pro gene therapy than myself. And I would encourage people to be involved in the trials because they’re looking for therapeutic help,” he said.
Encouraging all to support research, from natural history studies on up
Even for people with types of LGMD without gene therapies in development, Decker noted that participating in natural history studies — which track the evolution of a disease in the absence of treatment — can be invaluable for moving science forward.
“That data that they have … is very valuable for when it comes time for a treatment,” Decker said of these studies. Having data on what the disease’s progression can look like is especially important in LGMD, because it can vary widely from person to person, Decker noted. “They have to set baselines, and limb-girdle is probably one of the hardest diseases to set your baseline in,” he said.
Supporting research now is of particular importance, as policies are moving to cut funding for medical research and shrink the size of agencies like the NIH as part of an effort to improve government efficiency and reduce its spending. Decker thinks such policies will have severe consequences for the rare disease community.
“It’s going to be devastating. …. They’re going to save a few bucks, but I think there could be a lot of people that will die down the road,” he said.
The MDA has opened an online portal for people in the U.S. to easily contact their congressional representatives and voice their opinions about funding cuts to the NIH and other medical research. The MDA created a similar portal regarding proposed cuts and limitations to Medicaid, which provides health insurance for many Americans with neuromuscular diseases.
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