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Metabolomic Biomarkers of Myotonic Dystrophy
Sponsor: 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 myotonic dystrophy by examining a urine sample and a blood sample, and performing muscle function testing. The information that is gathered from this study may help to evaluate, prevent, diagnose, treat, and improve the understanding of human myotonic dystrophy.
Key Details
Gender
All
Age Range
14 Years - Any
Study Type
OBSERVATIONAL
Enrollment
64
Start Date
2026-10-26
Completion Date
2031-08
Last Updated
2026-11-03
Healthy Volunteers
Yes
Conditions
Locations (1)
Massachusetts General Hospital
Boston, Massachusetts, United States