2004-2006
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. |
| Results Available | Congenital myotonic dystrophy in a national registry (2010) |
| 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. |
| Results Available | Pregnancy and birth outcomes in women with FSHD (2006) |
| 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. |
| Results Available |
Chronic pain in persons with myotonic dystrophy and FSHD (2008) |
| 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. |
| Results Available | Open-label trial of recombinant human insulin-like growth factor 1/recombinant human insulin-like growth factor binding protein 3 in myotonic dystrophy type 1 (2011) More information |
| 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. |
| Results Available | The hypocretin neurotransmission system in myotonic dystrophy type 1 (2008) |
| 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. |