The Boy Who Got His Life Back Thanks to New Gene Therapy

The boy who got his life back thanks to new gene therapy

'It is nice to have my freedom back and not be in hospital anymore'

New analysis tracks Elevidys outcomes three years after treatment

Three years after receiving the gene therapy Elevidys (delandistrogene moxeparvovec-rokl) in a clinical trial, boys with Duchenne muscular dystrophy (DMD) are showing sustained improvements in physical function compared with what would be expected without treatment, according to new data announced by Sarepta Therapeutics, Elevidys’ developer. “ELEVIDYS is the first gene therapy for Duchenne to show a dramatic shift in disease trajectory out to t…

New 3-Year EMBARK Data Highlights Continued Therapeutic Effect of Gene Therapy Elevidys in Duchenne | NeurologyLive - Clinical Neurology News and Neurology Expert Insights

New findings from the EMBARK study highlight Elevidys' significant long-term benefits in slowing Duchenne muscular dystrophy progression in young patients.