(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
New data show pre-symptomatic babies with spinal muscular atrophy (SMA) treated with Evrysdi maintained the ability to swallow Evrysdi has demonstrated consistent clinically meaningful efficacy in adults, children, and babies two months and older and is now approved in 58 countries worldwide Further … Read the full press release
Posted in Healthcare, News, Pharma & Biotech, Science, Switzerland, Technology
Tagged DMD, Duchenne muscular dystrophy, Endeavor, Evrysdi, gene therapy, Hammersmith Infant Neurological Examination, HINE-2, Levi Garraway, M.D. Ph.D, motor milestones, neuromuscular, RAINBOWFISH study, risdiplam, Roche, Sarepta, SMA, spinal muscular atrophy, SRP-9001, World Muscle Society (WMS) Virtual Congress
Adults in the UK with Duchenne muscular dystrophy are being failed on critical medical checks that could lengthen their lives, according to research published by Newcastle University. Newcastle, UK, 22-1-2015 — /EuropaWire/ — A study across seven EU countries found that … Read the full press release
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