Sarepta Therapeutics announced positive topline data from the EMBARK trial, showing that Elevidys, 3 years post treatment, significantly slows disease progression on functional measures in ambulatory individuals who were 4-7 years old when treated. These 52 treated individuals, who participated in Part 1 of EMBARK and now are at a mean age of 9 years old, showed continuing benefits with a mean North Star Ambulatory Assessment (NSAA) above baseline, while an external control group continued to show the expected age-related decline below their baseline score. The treated individuals also showed significant slowing of progression on Time To Rise (TTR) and 10-Meter Walk Run (10MWR) compared to external controls.
Link to press release: https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-announces-positive-topline-three-year-embark-results
The post Sarepta reports positive 3-year data from the EMBARK trial: 4-7 year olds treated with their microdystrophin gene therapy, Elevidys, have significantly slower disease progression appeared first on CureDuchenne.