Research Studies, 2004-2006 Project title: Characteristics of Congenital Myotonic Dystrophy patients in a passive surveillance registry Investigator: Craig Campbell, MD FRCPC, Children’s Hospital of Western Ontario Approval: 04/2006 Study Type: Deidentified data Description: The aim of this study is to better understand the demographic and medical characteristics of Registry members diagnosed with Congenital Myotonic Dystrophy. This preliminary study may prepare for and encourage future research in the area of Congenital Myotonic Dystrophy. Project title: The Course and Outcome of Pregnancy and Delivery in Women with FSH Muscular Dystrophy Investigator: Emma Ciafaloni, MD, University of Rochester Approval: 12/2004 Study Type: Recruitment Description: Women completed surveys to increase the knowledge about the effects of pregnancy, delivery and post- partum recovery on the course of muscular dystrophy. The ultimate goal is to improve counseling, family planning and care of pregnant women with FSHD. Project title: Promotion of Health and Wellness Through Community Recreation and Exercise: Impact of Impairment, Pain, Self-Efficacy and Environmental Barriers Investigator: Craig McDonald, MD. University of California, Davis. Approval: 10/2004 Study Type: Recruitment Description: Volunteers for this study completed a detailed questionnaire asking about health status, physical activity, lifestyle issues, pain, nutrition, and diet. The goal is to better understand the factors that contribute to health, wellness and community integration in persons with slowly progressing neuromuscular diseases. Project title: Effects of SomatoKine in Myotonic Dystrophy Type 1 (DM1) Investigator: Richard T. Moxley III, MD, University of Rochester Approval: 9/2004 Study Type: Recruitment Description: This trial was performed to determine if SomatoKine is safe and well tolerated in subjects with DM1. Other goals were to determine whether SomatoKine improves muscle strength, function and muscle mass in subjects with DM1. Project title: The Pathophysiology of Hypersomnolence in Myotonic Dystrophy Investigator: Emma Ciafaloni, MD. University of Rochester Approval: 2/2004 Study Type: Recruitment Description: This project studied the differences between individuals with Myotonic Dystrophy and FSHD with regards to their tendency to fall asleep during different daytime activities. Project title: US Validation of a Neuromuscular Disease Specific, Individualized, Quality of Life Measure (The INQoL US Project) Investigator: Michael Rose, MD. King’s College Hospital, England Approval: 2/2004 Study Type: Recruitment Description: This project studied whether patients with neuromuscular diseases living in the US answer questions on a quality of life survey differently than patients with similar diagnoses who live in England.