Advocates say 2026 will be ‘an exciting time’ for DMD community
Approval expected this year for several new Duchenne MD therapies
For people with Duchenne muscular dystrophy (DMD), 2026 promises to be a year of tremendous excitement, with several new therapies poised for likely approval in the U.S., according to DMD advocates.
“It’s an exciting time,” Debra Miller, founder CEO of CureDuchenne, said in an interview with Muscular Dystrophy News Today. “A lot of hard work is finally coming to fruition now.”
CureDuchenne is an advocacy organization dedicated to supporting people with DMD and accelerating research. Miller and her husband Paul founded CureDuchenne in 2003 after their son was diagnosed with DMD.
In the more than 20 years since, CureDuchenne has helped to fund the development of many different treatment approaches for DMD — and those investments are starting to pay off, Miller noted, as more and more promising treatments are expected to be approved this year and in those ahead.
Michael Kelly, PhD, CureDuchenne’s chief scientific officer, is also optimistic about the coming months.
“I think this year is going to be particularly exciting,” Kelly said. “A lot of hard work has gone in over the last decade, … in order to really get the field into a position where it is today.”
DMD is caused by mutations in the gene that encodes dystrophin, a protein that’s essential for muscle health. Lacking dystrophin, muscles accumulate damage over time, leading to disease symptoms such as muscle weakness and wasting.
In the decades since CureDuchenne’s founding, scientific understanding of DMD has expanded dramatically, which has helped in the development of new treatments.
“We’re just really proud to have been a part of the acceleration of meaningful treatments,” Miller said. “And when I say accelerate, what used to take 10 years seems like it’s taking two years now. We’ve learned an awful lot about the disease, about the science, about endpoints and clinical trial design and what regulators need to see, the patient population — and because of all that work that we put in early, we now are able to see these … development programs move much quicker.”
Scientists already working to make gene therapies safer
Several cutting-edge therapeutic strategies for DMD aim to treat the disease by addressing the underlying deficiency of dystrophin protein. Kelly said that such approaches will be “foundational” for the treatment of DMD. “The disease is caused by a lack of [the dystrophin protein], so it’s clear that we want to put … some form of dystrophin back in as a primary method of attacking the disease,” he said.
One dystrophin-targeting treatment approach for DMD is gene therapy, which aims to deliver a gene encoding a healthy version of the dystrophin protein to muscle cells. The first gene therapy for DMD, Sarepta Therapeutics‘ Elevidys (delandistrogene moxeparvovec-rokl), was conditionally approved by the U.S. Food and Drug Administration (FDA) in 2023 for certain patients who can walk. The next year, the FDA gave the therapy full approval for ambulatory patients ages 4 and older, as well as conditional approval for nonambulatory patients in the same age range.
But then in 2025, two nonambulatory patients died of liver complications after receiving Elevidys. Following these deaths, the FDA revoked the conditional authorization for patients who cannot walk. Kelly said these developments were disappointing, but not altogether unexpected — the DMD community has long been aware that gene therapy carries risks, he said, noting that some patients died during clinical testing of an early DMD gene therapy candidate that was ultimately discontinued.
And Kelly expects that gene therapy will still play a key role in DMD treatment in the future. He noted that Sarepta is already working on protocols to make Elevidys safer, and meanwhile, other gene therapies are in development, including Regenxbio‘s RGX-202. That gene therapy has shown promise in early clinical testing, and Regenxbio is planning this year to ask the FDA to grant RGX-202 conditional approval. Kelly said he expects RGX-202 will be approved by early 2027.
Goal is to have treatments for all those affected by DMD
Another DMD treatment strategy that targets dystrophin is exon skipping, which basically aims to get cells to skip over part of the mutated gene to allow the production of a shortened but functional dystrophin protein. Several exon-skipping therapies are conditionally approved by the FDA. These conditional approvals were based on early data showing the therapies boosted dystrophin levels, but continued approval is contingent on further tests to confirm clinical benefit.
Sarepta recently announced that the Phase 3 ESSENCE trial (NCT02500381), which tested the exon-skipping therapies Amondys 45 (casimersen) and Vyondys 53 (golodirsen), failed to meet its goal of showing the therapy was better than a placebo at improving the time it took patients to climb four steps. Kelly said this result was disappointing, but he stressed that Amondys 45 and Vyondys 53 were some of the first exon-skipping therapies ever to be developed — and as is often the case in drug development, these forerunners aren’t as potent as newer medicines.
“We always knew that their chemistry that [Amondys 45 and Vyondys 53 were] not the most efficacious drugs that were around,” Kelly said. “They were important whenever they were first approved, but they’ve since been superseded, I feel, by newer technologies.”
Most of that has occurred just recently, according to Kelly.
“We’ve witnessed the whole exon skipping field mature dramatically over the last couple of years,” Kelly said, adding that he and other scientists have “real high expectations” for the next generation of treatments in this class “that are going to supersede or surpass all of the results that we’ve seen for the older generation of drugs.”
Kelly highlighted a few promising exon-skipping therapies that he expects to win conditional FDA approval in the coming year or so, including Avidity Biosciences‘ delpacibart zotadirsen and Dyne Therapeutics‘ DYNE-251. Miller noted that, although each of these therapies is designed to treat only patients with certain mutations, the success of any one therapy “gives us the confidence and the proof of concept that we can make it happen” for all patients. Avidity, Miller added, has such confidence in delpacibart zotadirsen that it made the therapy available to all U.S. patients amenable to exon 44 skipping under a managed access program.
Researchers in Duchenne MD looking beyond dystrophin protein
Beyond gene therapy and exon-skipping therapies, other DMD treatment strategies aim at targets other than the dystrophin protein. For example, Capricor Therapeutics is developing deramiocel, also known as CAP-1002, an experimental therapy that uses immature heart cells that secrete signaling molecules to promote muscle repair. The company recently announced that the Phase 3 HOPE-3 clinical trial (NCT05126758) had hit its main goal, with deramiocel outperforming a placebo at preserving arm and heart function.
The FDA rejected a previous application seeking approval of deramiocel based on earlier Phase 2 study results, saying more data were needed to prove the therapy is effective. Now Capricor is planning a resubmission in hopes these new Phase 3 data will help convince the FDA to grant approval, and Kelly said he expects the cell therapy to be approved in the U.S. before the end of the year.
We see combination therapy as [having] an important role. … As [new] drugs are rolled out, patients are going to have multiple opportunities to think about what is the best and most suitable combination for them, in addition to [using] just one therapy.
Kelly noted that deramiocel showed benefits even in patients who had also been treated with gene therapy or exon-skipping medicines. “So it’s going to be a standalone, but it’s also going to be a really important combination drug,” he said. Kelly and Miller both agreed that combinations of therapies, including medicines that target dystrophin and treatments with other mechanisms, will be important for DMD patients in the future.
“We see combination therapy as [having] an important role,” Kelly said. “As these drugs are rolled out, patients are going to have multiple opportunities to think about what is the best and most suitable combination for them, in addition to [using] just one therapy.”
As the field of DMD treatment continues to evolve, CureDuchenne will continue pushing for research advances and providing support to affected families.
Miller highlighted the organization’s one-to-one program, where families can have one-on-one discussions with DMD experts. “We can’t give medical advice, but we can definitely help the families understand the landscape of what’s available and what might be appropriate for their child,” she said.
On the treatment front, Kelly said, “Our goals are really to go beyond where we are today.” He added: “That’s what’s going to continue over the next two to three years.”
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