Duchenne muscular dystrophy — sometimes referred to simply as Duchenne — is a genetic disorder that causes progressive muscle loss and weakness. It occurs as a result of gene mutations involved in the production of a protein called dystrophin, according to the Muscular Dystrophy Association (MDA).
Data presented by Barry Byrne, MD, Ph.D. suggest that intravenous SGT-001 (Solid Biosciences) is well-tolerated and efficacious in DMD.
Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy, awarded the University of Florida Wellstone Muscular Dystrophy Cooperative Center a grant for $348,891.
A new five-year, $6.25 million grant from the National Institutes of Health will support the University of Florida’s continued efforts to develop MRI biomarkers for individuals with Duchenne muscular dystrophy, and expand the study to include people with Becker muscular dystrophy.