Dr. Matt Disney and Dr. John Day provide an overview of the DM2 disease mechanism and describe how compounds can be designed as potential therapies for this disease.
Dr. Bruce Wentworth, Ph.D., of Genzyme, presents an overview of ASO treatments, describing in lay language how they are intended to treat DM.
Dr. Charles Thornton, M.D., of the University of Rochester, discusses the infrastructure, standards and other components necessary to support successful clinical studies and trials, and the status of developing those components for DM trials.
Join a new generation's fight against muscle disease!
As the inaugural presentation in our Living with DM Webinar series, we discuss the decline of executive function in the CNS due to myotonic dystrophy, as well as strategies for managing this symptom, and an update on current CNS research.