This contract establishes a National Registry for Myotonic Dystrophy and Facioscapulohumeral Dystrophy Patients and Families. Myotonic dystrophy (DM) and Facioscapulohumeral Muscular Dystrophy (FSHD) are the most common forms of adult muscular dystrophy and both cause progressive, disabling weakness, and sometimes cause sudden death. The long term goal for the Registry is to facilitate research in DM and FSHD by functioning as a synergistic liaison between families afflicted by these diseases, who are eager to participate in research, and the investigators interested in studying these disorders. The Registry has a multi discipline Scientific Advisory Committee that will establish diagnostic criteria for DM and FSHD, including specific clinical definitions to diagnose patients who do not have DNA proven DM or FSHD. The Registry will solicit, recruit, classify patients, and store medical and family history data for patients with clinically diagnosed DM and FSHD and facilitate research by providing to investigators statistical analysis of Registry data and answers to specific questions about the data; and, by providing for investigators a means to contact individuals and families who have given their consent and are eager to participate in their specific research studies. The Scientific Advisory Committee monitors the function of the Registry, assesses the value of proposed clinical studies, and to reviews all proposals submitted by investigators who wish to obtain the names of Registry members or accessibility of Registry data for research projects.
