Individual with Duchenne dies from acute liver failure after gene therapy treatment

Sarepta Therapeutics has shared that a young man with Duchenne passed away following treatment with ELEVIDYS, an AAV-delivered micro-dystrophin gene therapy.  Sarepta has shared that the individual suffered from acute liver failure after treatment, and will provide more information as they further gather and analyze the data.

This disease is unrelenting and new therapeutics are necessary, but can come with risks.  We are saddened for the Duchenne community, and our thoughts and prayers go out to the family and friends of this young man.