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The Myotonic Dystrophy Family Registry (MDFR) is an online, patient-entered database that collects information on myotonic dystrophy (DM) to aid researchers in developing new, effective treatments and help identify participants for research studies and clinical trials.
The Myotonic Dystrophy Family Registry (MDFR) is an online, patient-entered database that collects information on myotonic dystrophy (DM) such as disease symptoms and demographic information to aid researchers in developing new, effective treatments and help identify participants for research studies and clinical trials.
The Registry supports trials and studies, making it easier for researchers to explore data and identify possible trial and study participants. It is the first DM registry that gives community members the opportunity to explore anonymous Registry data, to see what the DM community looks like and what others with DM experience. It also provides information on the community of people living with DM, giving researchers and other medical professionals the opportunity to improve how they treat those affected with DM and learn more about how and why certain treatments work and don't work.
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Patient-entered data | Other | This registry provides patient-entered data for future clinical trial and study use. |
| Measure | Description | Time Frame |
|---|---|---|
| Patient reported outcomes | Number of patients reporting specific symptoms and symptom severity, as well as impacts to quality of life and overall burden of disease in order to inform clinical trial development, understanding of disease for academic, industry and federal agency stakeholders and overall policy decisions. Results will be analyzed in comparison to other registry data and surveys to characterize this disease population cohort and to further define the population. | 36 months |
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Inclusion Criteria:
Exclusion Criteria:
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Participants will volunteer to participate in this study and enter their own information. The study will be advertised through neuromuscular disease clinics and the Myotonic Dystrophy Foundation and Myotonic Dystrophy Family Registry websites.
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Sofia Olmos, PhD | Contact | 415-800-7777 | coordinator@myotonicregistry.org |
| Name | Affiliation | Role |
|---|---|---|
| Tanya Stevenson, EdD, MPH | Myotonic Dystrophy Foundation | Study Chair |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Myotonic Dystrophy Foundation | Recruiting | Oakland | California | 94612 | United States |
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| Label | URL |
|---|---|
| Myotonic Dystrophy Family Registry (MDFR) Web portal. | View source |
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| ID | Term |
|---|---|
| D009223 | Myotonic Dystrophy |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
| D020967 | Myotonic Disorders |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D009422 | Nervous System Diseases |
| D009468 | Neuromuscular Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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