A gene therapy for Duchenne muscular dystrophy (DMD) was paused after safety concerns, including three deaths. The drug — delandistrogene moxeparvovec-rokl, also known as Elevidys — is back on the market, but only for some people and with new safety warnings.

Elevidys is a one-time gene therapy developed by Sarepta Therapeutics. The treatment helps the body make a shortened form of dystrophin, a protein that keeps muscles strong. People with DMD don’t make enough of this protein, which causes their muscles to weaken over time.

The FDA first granted accelerated approval to Elevidys in June 2023 for children with DMD who are nonambulatory (use wheelchairs). In June 2024, the agency granted full approval for children ages 4 and older who are ambulatory (able to walk).

In 2025, after three people — including two teens with DMD — died from liver failure, the FDA asked Sarepta to pause all shipments of Elevidys and put related clinical trials on hold. At first, the company continued to provide the drug for ambulatory children. But on July 22, Sarepta voluntarily paused all shipments to work with the FDA on new safety labeling for the drug.

Soon after, the FDA allowed Sarepta to restart shipments for ambulatory children. While safety studies continue, the pause is still in place for those who are nonambulatory.

Safety Concerns Trigger a Temporary Market Withdrawal

While Elevidys’ shipments were on hold, Sarepta released new safety data in August 2025. The data showed that liver problems were more common than expected, especially in children who use wheelchairs. In clinical trials, nearly half (46.5 percent) of participants had signs of liver injury, and 5 percent were hospitalized. In real-world use, 8.4 percent of nonambulatory people were hospitalized for liver issues, compared with 5.8 percent of those who were ambulatory.

As part of restarting shipments to ambulatory children, the FDA required updates to Elevidys’ safety labeling, including a black box warning to highlight the risk of acute liver injury (sudden, serious liver damage). People receiving Elevidys must have regular liver and heart monitoring and take corticosteroids to help reduce inflammation. The drug is no longer recommended for people with certain genetic mutations (changes) or high levels of anti-AAV antibodies (proteins the body makes that can block the therapy).

Sarepta also plans to test whether an immunosuppressant (sirolimus) given before Elevidys can lower the risk of liver damage. Results from this small study are expected in the first half of 2026.

What This Means for People Living With DMD

Elevidys is currently approved only for ambulatory children who have a confirmed DMD gene mutation. The FDA has paused its use in nonambulatory individuals until more safety data is available. This is a cautionary moment for gene therapy.

For people living with DMD, Elevidys still holds promise, but the path forward is uncertain. As with all treatments, especially newer ones, it’s important to carefully weigh the potential benefits and risks with your healthcare provider. If you’re considering gene therapy, talk with your neurologist or care team about:

• Whether you or your child meets the current eligibility criteria
• What kind of monitoring and follow-up care would be needed
• How liver health and the body’s immune response might affect safety

Learn more about gene therapy and other treatments for DMD.

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