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Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1) Extension
Sponsor: Virginia Commonwealth University
Summary
Myotonic Dystrophy type 1 (DM1) is an autosomal dominant multisystemic disorder that causes progressive disability and shortened life expectancy. It is characterized by progressive weakness and myotonia, which preferentially affects the craniofacial, hand, and distal leg muscles. Many patients also experience difficulties with cognition, cardiac arrhythmias, respiratory failure, or cataracts. Currently there is no treatment to slow progression or reverse the symptoms.
Key Details
Gender
All
Age Range
18 Years - 70 Years
Study Type
OBSERVATIONAL
Enrollment
1000
Start Date
2026-07
Completion Date
2032-12
Last Updated
2026-07-27
Healthy Volunteers
No
Conditions
Locations (1)
Virginia Commonwealth University
Richmond, Virginia, United States