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.
Dr. Tom Cooper, MD, of Baylor College of Medicine, reports on the International Myotonic Dystrophy Consortium (IDMC), a biennial meeting held in San Sebastian, Spain, just prior to the conference.
Dr. Charles Thornton describes the remarkable advances achieved in myotonic dystrophy research and the implications of that progress for the state of research funding overall.