Cardiotoxic Effects and Microdystrophin Expression after Gene Therapy for DMD

“While AAV itself does not cause disease in humans, systemic delivery at the high doses that are needed to reach all muscles carries immune-mediated and organ-specific toxicities. In recent years there have been hospitalisations, acute-liver injury events, myocarditis, and a small number of fatalities associated with AAV therapies, including for DMD.”

“Delivering state-of-the-art DMD gene therapy requires a multidisciplinary team working in a tightly integrated system to optimize safety monitoring, interpret complex signals and manage side effects effectively.”