Longtime MDA research grantee honored for work in LGMD gene therapy
A team headed by neurologist Jerry Mendell, a longtime MDA research grantee and director of the MDA Clinic at Nationwide Children's Hospital in Columbus, Ohio, has received the prestigious Annals of Neurology prize for an outstanding contribution to clinical neuroscience.
Five of six people with limb-girdle muscular dystrophy type 2D produced protein from the new genes
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...
An MDA grant will move researchers toward a clinical trial involving bloodstream delivery of the alpha-sarcoglycan gene to thigh muscles.
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...