Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1) Extension

Recruiting Observational Study
DM1 Myotonic Dystrophy Myotonic Dystrophy 1 Myotonic Dystrophy Type 1 Myotonic Dystrophy Type-1 Myotonic Dystrophy, Type 1 (DM1) Myotonic Muscular Dystrophy
No Placebo Group Every participant receives an active treatment — no one gets a placebo. No Study Drug Researchers observe your health over time — no experimental treatment is given.
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At a Glance
Age
18 – 70
Sex
Any
Study type
Observational
Participants needed
1,000 (estimated)
Sponsor
Virginia Commonwealth University · Other
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About This Trial
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. Curr…
Trial Locations
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Eligibility Criteria
Inclusion Criteria: * Age 18 to 70 years (inclusive) * Written, voluntary informed consent must be obtained prior to any study procedures. In cases where a Legally Authorized Representative (LAR) provides consent, verbal assent will be obtained from the subject, as determined by the investigator an…
Contacts

Jennifer Raymond

804-828-6318

Jennifer.raymond@vcuhealth.org

CONTACT

Ruby Langeslay

804-828-6318

Ruby.langeslay@vcuhealth.org

CONTACT