Five of six people with limb-girdle muscular dystrophy type 2D produced protein from the new genes

posted on October 29, 2010 - 2:25pm
Results from an MDA-supported, phase 1 study of gene therapy for the type 2D form of limb-girdle muscular dystrophy (LGMD2D) show sustained protein production from the transferred genes in two out of three trial participants six months after injection of the genes into a foot muscle. The new results follow an announcement last year showing protein production from transferred genes in the first...

"This Month in Muscular Dystrophy" is the subject of a new audio series from Nationwide Children's Hospital in Columbus, Ohio

posted on May 24, 2010 - 3:53pm
Scientists at the Neuromuscular Disorders Program at Nationwide Children’s Hospital in Columbus, Ohio, are producing a series of podcasts examining current research in neuromuscular disease. The series, “This Month in Muscular Dystrophy,” is an opportunity to hear authors of recent publications discuss how their work improves understanding of these diseases, and what it might mean for treatment.

An MDA grant will move researchers toward a clinical trial involving bloodstream delivery of the alpha-sarcoglycan gene to thigh  muscles.

posted on December 23, 2009 - 5:11pm
Development of delivery of a therapeutic gene via the bloodstream to the thigh muscles in people with type 2D limb-girdle muscular dystrophy (LGMD2D) is proceeding on schedule, thanks in part to a new $458,814 grant from MDA to neurologist Jerry Mendell at Nationwide Children's Hospital in Columbus, Ohio. MDA's Board of Directors approved the new funding, via the Association's translational...