Dr. Darren Monckton explains the current research concerning the significance of CTG repeats for persons with DM1.
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.
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