Two Children Have Died After Taking a Duchenne Drug. What Went Wrong? – by Sean C. Domnick
Another child has died after receiving Elevidys, a gene therapy for Duchenne muscular dystrophy. This is the second reported death tied to the treatment—and it raises troubling questions about how the drug was approved, how it’s being used, and whether the proper safeguards were in place to protect the most vulnerable patients.
Elevidys, developed by Sarepta Therapeutics, was approved last year by the Food and Drug Administration for children over the age of four who could still walk. But the company soon began administering the drug to children who had already lost that ability—a group that had not been adequately studied in clinical trials.
Now, in the wake of two fatalities among these non-ambulatory patients, Sarepta has announced it will pause the drug’s use in this population while it reassesses the risks. In other words, research that arguably should have been completed before the drug was given to these children is now being done after the fact.
Warnings Existed. But Were They Enough?
Both children who died were non-ambulatory. The full details have not yet been made public, but liver damage is a major concern. Elevidys already carries a warning label noting the risk of liver injury. In these recent cases, it’s possible that liver failure occurred—something that would far exceed what regulators and clinicians expected.
In one of the deaths, the child also had a cytomegalovirus (CMV) infection, which can affect the liver. But that doesn’t erase the underlying issue: the safety of systemic gene therapy, particularly in children whose bodies are already under immense stress, is still not fully understood. And when the science is uncertain, caution must lead the way.
Even the FDA Is Divided
What makes this case especially concerning is that even the FDA appears to have had internal doubts. The head of its gene therapy division publicly questioned whether Elevidys should have been approved at all—a striking statement from someone inside the agency charged with protecting public health.
For families affected by Duchenne muscular dystrophy, the drug represented a rare glimmer of hope. The disease is progressive and terminal, and the urgency to find effective treatments is entirely understandable. But speed can’t come at the expense of safety. Regulatory processes exist for a reason, especially when the patients involved are children.
Gene therapy is one of the most promising frontiers in medicine. But it’s also one of the riskiest. These treatments involve altering DNA and often rely on viral vectors to deliver genetic material into the body. The long-term effects are not always predictable, and even short-term risks, like organ toxicity, can be devastating.
What Happens Now
There is still much we don’t know about what went wrong. Investigations are ongoing, and Sarepta has said it is working to understand the causes of the deaths. But in the meantime, families, clinicians and policymakers should be asking difficult but necessary questions.
What was the basis for expanding the drug’s use to a patient group that had not been part of the original approval? Were doctors and families fully informed of the potential risks? And does this point to a larger problem in how new therapies are being fast-tracked through regulatory systems?
These are not academic concerns. They are questions with life-and-death consequences.
Medical innovation is essential, but so is humility—especially when dealing with diseases we do not fully understand and treatments that carry uncertain consequences. The tragic deaths of these two children must not be written off as isolated or unfortunate events. They should serve as a warning: when we lower the bar for evidence, vulnerable patients pay the price.
About Sean C. Domnick
Sean C. Domnick is a Shareholder at Rafferty Domnick Cunningham & Yaffa, a preeminent national boutique law firm based in Palm Beach Gardens and Pensacola, Florida.
