(IN BRIEF) Roche has reported positive results from the second year of the EMBARK Phase III trial of Elevidys (delandistrogene moxeparvovec), the first approved gene therapy for Duchenne muscular dystrophy (DMD). The results showed statistically significant and clinically meaningful improvements … Read the full press release →
Posted in Healthcare, Pharma & Biotech, Science, Technology
Tagged 10-meter walk/run, 10MWR, clinical data, clinical efficacy, crossover group, delandistrogene moxeparvovec, disease-modifying treatment, Duchenne muscular dystrophy, Elevidys, EMBARK trial, external control group, functional improvements, gene therapy, micro-dystrophin, motor function, muscle biopsies, muscle function preservation, muscle pathology, North Star Ambulatory Assessment, NSAA, Phase III study, regulatory approvals, Roche, safety profile, Sarepta Therapeutics, Time to Rise, TTR
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