Roche on Monday revealed positive results from the EMBARK trial, showing that its gene therapy Elevidys has sustained benefits for individuals with Duchenne muscular dystrophy (DMD) over a two-year period.
The phase III study demonstrated significant improvements in motor functions, such as standing, walking, and running, compared to an untreated control group.
The trial evaluated Elevidys, also known as delandistrogene moxeparvovec, in ambulatory individuals with DMD. The study's findings highlighted statistically significant and clinically meaningful improvements across three key functional outcomes: the North Star Ambulatory Assessment (NSAA), Time to Rise (TTR), and the 10-meter walk/run (10MWR).
Notably, these benefits not only persisted but increased from the first to the second year after treatment.
Roche also reported that no new safety concerns were identified, reinforcing Elevidys's consistent and manageable safety profile observed to date.
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