We are deeply saddened by the loss of a young member of our community following treatment with ELEVIDYS. According to Sarepta, the individual with Duchenne received ELEVIDYS and later suffered from complications of acute liver failure.

This represents the first reported case of acute liver failure resulting in death following treatment with ELEVIDYS. According to Sarepta, while acute liver injury is a known risk of ELEVIDYS and other AAV-mediated gene therapies, this level of severity had not been observed in the more than 800 patients treated across clinical trials and commercial use. In a letter to the community, Sarepta indicated that they have reported this event to regulatory agencies, informed prescribing clinicians, are actively assessing any potential contributing factors, and will continue monitoring and updating prescribing information as more details emerge.

Our hearts ache for this family, and this tragic loss highlights the urgent need to fully understand the risks associated with gene therapy in our current therapeutic landscape to ensure patient safety. As we mourn, it is imperative that we also reaffirm our commitment to working together—families, researchers, clinicians, industry partners, and regulatory agencies—to strengthen safety protocols, enhance monitoring, and advocate for the well-being of every individual receiving treatment. 

Our community stands united, determined to make Duchenne therapies as safe and effective as possible. Families that may have questions or concerns about this news should consult with their clinicians, who can provide individual guidance.  

A Note From PPMD’s Pat Furlong

Our hearts are heavy today. The loss of a child is a tragedy beyond words, and our entire community grieves alongside this family and their loved ones. Let us lean on one another in this time of mourning as we grieve, seek understanding, and push forward with hope.

We recognize the courage it takes for families to pursue new treatment options in the fight against Duchenne. At PPMD and as a community, we remain committed to advocating for transparency, safety, and ongoing research to ensure that every individual has the information they need to make informed decisions. 

To the family and friends of this beloved young man, please know that you are not alone. Your loss is deeply felt, and we are here to offer our unwavering support and comfort during this difficult time.