– TIME recognizes Dr. Mendell’s decades-long contributions in the treatment of neuromuscular disease and the advancement of gene therapy, including the first gene therapy for Duchenne muscular.
/PRNewswire/ On February 8, 2024, The Speak Foundation, a patient-led 501(c)(3) organization for Limb-Girdle Muscular Dystrophy (LGMD), will be convening a.
Delandistrogene moxeparvovec improved motor function and was well-tolerated 4 years after treatment in a small cohort of children with Duchenne muscular dystrophy, according to a poster at the 2023 MDA Clinical & Scientific Conference.“Delandistrogene moxeparvovec is an investigational gene transfer therapy developed to address the root cause of Duchenne muscular dystrophy through