PALM BEACH GARDENS, Fla., June 24, 2025 /PRNewswire/ -- A second child has died after receiving Elevidys, an experimental gene therapy for Duchenne muscular dystrophy—raising serious concerns about regulatory shortcuts, patient safety, and the urgent need for oversight.

In a new article, Florida-based trial attorney Sean C. Domnick of Rafferty Domnick Cunningham & Yaffa exposes the troubling failures that led to these deaths and questions how a drug approved for a narrow group of patients was expanded to include children who had never been studied in clinical trials. Elevidys, manufactured by Sarepta Therapeutics, was fast-tracked by the FDA last year for ambulatory children over age four. But the company began administering the drug to non-ambulatory patients—a population with significantly different risks.

Now, two of those children are dead.

"Medical innovation is essential—but so is humility," Domnick writes. "When we lower the bar for evidence, vulnerable patients pay the price."

The deaths have raised red flags across the medical community. Sarepta has since paused use of the therapy in non-ambulatory patients, and an internal review is underway. Reports suggest liver failure may have been a contributing factor—despite existing warning labels and a known risk of liver injury with this therapy.

Even the FDA's own gene therapy division reportedly had internal doubts about the drug's approval, underscoring concerns that critical safeguards were overlooked in the rush to market.

As Domnick explains, the situation reflects broader systemic issues—particularly around how the FDA handles fast-track approvals and whether families are truly informed of the risks.

"These are not isolated incidents. They're a warning," he said. "We need to reexamine how therapies are being pushed through before we fully understand their consequences—especially for children."

With families desperate for solutions and biotech companies racing to innovate, the balance between speed and safety has never been more critical. Sean Domnick and the team at Rafferty Domnick Cunningham & Yaffa are calling for increased transparency, stronger informed consent practices, and stricter enforcement of clinical trial protocols before vulnerable patients are exposed to unproven treatments.

About Rafferty Domnick Cunningham & Yaffa 
Rafferty Domnick Cunningham & Yaffa is a plaintiff litigation firm with a national practice specializing in catastrophic injury and mass torts law. With a team of experienced attorneys dedicated to fighting for the rights of those injured through negligence or wrongdoing, Rafferty Domnick Cunningham & Yaffa has earned a reputation for delivering justice and securing substantial settlements for their clients.

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