Tag: Duchenne muscular dystrophy treatments

Jerry R. Mendell, MD, Receives 2023 Best Abstract Award for DMD Gene Therapy Research

sarah JonasAnnouncement

The American Neuromuscular Foundation, is excited to announce Jerry R. Mendell, MD, as the recipient of the 2023 Best Abstract Award, for his abstract titled, “Long-Term Safety and Efficacy in Patients With Duchenne Muscular Dystrophy 4 Years Post-Treatment With Delandistrogene Moxeparvovec in a Phase 1/2a Study.”

Scientists uncover a novel approach to treating Duchenne muscular dystrophy

sarah JonasResearch Results

Scientists at Sanford Burnham Prebys, Fondazione Santa Lucia IRCCS, and Università Cattolica del Sacro Cuore in Rome have shown that pharmacological (drug) correction of the content of extracellular vesicles released within dystrophic muscles can restore their ability to regenerate muscle and prevent muscle scarring. The study, published in EMBO Reports, reveals a promising new therapeutic approach for Duchenne muscular dystrophy (DMD), an incurable muscle-wasting condition.