Gaining a better understanding of the cause of two deaths linked to dilandistrogene moxeparvovec in order to better prevent them

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US experts in gene therapy for Duchenne muscular dystrophy (DMD) have investigated the causes of two deaths that occurred in 2025 and were attributed to the administration of a single dose of dilandistrogene moxeparvovec (Elevidys®). They propose the following preventive measures:

  • the two patients concerned were aged 15 and 16 respectively and had no risk factors other than their DMD,
  • the first symptoms appeared four weeks after the initial infusion and manifested as elevated transaminases, followed by full-blown hepatocellular failure leading to death,
  • The authors emphasise the warning and adverse nature of the sharp rise in bilirubin levels, even as liver enzyme levels were tending to normalise;
  • pre-injection liver function tests will now need to be more comprehensive and more frequent in order to identify patients at risk;
  • However, there is no consensus on whether or not it is necessary to prevent this type of adverse event by using a high-dose immunosuppressive regimen (such as sirolimus) prior to gene therapy.

 

Learnings from Patient Mortality after Delandistrogene Moxeparvovec Administration: A Report of Two Cases and Expert Committee Considerations for Future Mitigation and Management. Shieh PB, Proud C, Diamond T et al. Hum Gene Ther. 2026 Mar

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