Extracellular RNA Biomarkers of Myotonic Dystrophy
Massachusetts General Hospital
Summary
Current methods of measuring the response to new treatments for muscular dystrophies involve the examination of small pieces of muscle tissue called biopsies. The investigators are interested in finding less invasive methods that reduce the need for muscle biopsies. The purpose of this research is to learn about the possibility of detecting and measuring the activity and severity of muscular dystrophies by examining a urine sample and a blood sample.
Eligibility
- Age range
- 5+ years
- Sex
- All
- Healthy volunteers
- Yes
Inclusion Criteria: * Subjects with DM1 or DM2 based on genetic testing and/or clinical criteria (some subjects who have positive genetic testing may be asymptomatic, while other subjects who show characteristic clinical features may have declined to have genetic testing done). Control non-DM subjects are unknown to have DM or any other muscular dystrophy by history and may have had no genetic testing. * Able to provide informed consent or assent for participation in the study. * Demographic characteristics for single biofluid collection: Males and females age 5 years and older (DM1, DM2, and…
Locations (3)
- Massachusetts General HospitalBoston, Massachusetts
- Beth Israel Deaconess Medical CenterBoston, Massachusetts
- University of Texas SouthwesternDallas, Texas