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.
Age range
18 Years – 70 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
Characterize the long-term disease progression- 10 meter walk/run
Timeframe: Baseline (0 months), every 12 months over four years
Characterize the long-term disease progression- vHOT
Timeframe: Baseline (0 months), every 12 months over four years
Characterize the long-term disease progression- grip strength
Timeframe: Baseline (0 months), every 12 months over four years
Characterize the long-term disease progression- ECG
Timeframe: Baseline (0 months), every 12 months over four years
Characterize the long-term disease progression- FVC
Timeframe: Baseline (0 months), every 12 months over four years
Characterize the long-term disease progression- DM1-Activ-c
Timeframe: Baseline (0 months), every 12 months over four years