MDF honored both Senator Dick Durbin and Senator Diane Feinstein with MDF Congressional Leadership Awards for their outstanding work and support in getting myotonic dystrophy added as an eligible research area in the U.S. Department of Defense (DOD) Peer-Reviewed Medical Research Program (PRMRP) for Fiscal Year 2018 (FY18).
Intronic GC-rich microsatellite expansions are common in neurological disorders and act to trigger the intronic retention that underlies disease pathogenesis.
Some of you may have watched the news show, 60 Minutes, on April 29th. The 60 Minutes piece of interest to everyone with an inherited disease, like DM, was titled: “CRISPR: The Gene-Editing Tool Revolutionizing Biomedical Research”.
When cardiologist Dr. William J. Groh examined a young woman with an arrhythmia in 1995, he thought it was unusual. She did not have known heart disease or heart failure that would normally be associated with a serious heart rhythm disturbance. What she did have, though, was myotonic dystrophy (DM).