New results three years following the phase 3 EMBARK clinical trial investigating Sarepta Therapeutic’s ELEVIDYS (gene therapy) brings encouraging news for families affected by Duchenne muscular dystrophy (DMD).
Three years after treatment, ambulatory boys with DMD between 4-7 years of age who received ELEVIDYS were able to keep higher levels of motor function compared to boys with DMD of similar age who did not receive the therapy. Their scores on the North Star Ambulatory Assessment (NSAA), a common measure of walking and movement, stayed above where they were before treatment. In comparison, ambulatory boys in the untreated group showed a decline consistent with the natural history of Duchenne.
The study showed that ELEVIDYS slowed the progression of DMD on key movement measures. Specifically, results show a slowing of disease progression by 73% in time-to-rise and 70% in the 10-meter walk-run measurement. Even more reassuring for families is that the benefits remained after three years suggesting that the therapy continues to help protect muscle function.
“ELEVIDYS is the first gene therapy for DMD to show a dramatic shift in disease trajectory out to three years consistent with earlier long-term data extending up to five years. This is long-term data in a robust, controlled clinical dataset that demonstrates the power of a disease-modifying therapy targeting the underlying cause of DMD.”
Louise Rodino-Klapac, Ph.D., president of research & development and technical operations, Sarepta.
Slowing disease progression may mean more time walking independently, greater ease with everyday activities, and a longer period of physical stability during childhood. While DMD remains a serious and progressive condition, these findings suggest that gene therapy may change its course, not just for months, but for years. Researchers will continue to follow boys who were part of this study to better understand long-term outcomes. The DMD community will be watching closely as this growing body of evidence helps shape future care and treatment decisions.
Want to know more about Duchenne Research?
Scientists and medical experts have been tirelessly pursuing diverse approaches to defeat Duchenne muscular dystrophy. From tackling the root cause to alleviating symptoms, the field of research is vast and promising. Explore the various strategies being developed and tested, and gain insights into the future of muscular dystrophy treatment: