Results in non-ambulatory people
Study 2 included non-ambulatory participants and was designed to measure the amount of ELEVIDYS micro-dystrophin produced and safety. The change in micro-dystrophin levels is shared below, and safety information from all trials can be found here.
A different study is measuring the impact on muscle function, but these results are not available yet.
Average increase in ELEVIDYS micro-dystrophin 3 months after treatment
Across all trials, ambulatory participants produced an average of 34% to 51% ELEVIDYS micro-dystrophin. Non-ambulatory participants produced an average of 40% ELEVIDYS micro-dystrophin.
STUDY 2
8 participants
People with Duchenne who are untreated typically have little to no dystrophin.
These results were consistent with what was seen in ambulatory participants. Results were measured using a test called western blot, which looks at a small piece of muscle from a person’s body to see if micro-dystrophin was produced. The numbers reflect the amount produced if 100% was the typical level in a person without Duchenne.
ELEVIDYS micro-dystrophin had a demonstrated impact on motor function in ambulatory people. While motor function data is not yet available for non-ambulatory people, ELEVIDYS was approved under accelerated approval based on the FDA’s belief that increased micro-dystrophin is reasonably likely to have a clinical benefit. More information is needed to confirm if there is a clinical benefit in non-ambulatory people. A clinical trial is ongoing to further evaluate ELEVIDYS in the non-ambulatory population. Safety information in non-ambulatory people is limited to 8 clinical trial participants.
Next up: Explore safety
Now that you’ve seen the results from the clinical trials, learn more about safety, including possible side effects.