Investigational treatment for adult-onset myotonic dystrophy type 1
Part of Bones, joints & muscles, Brain & nervous system, Genetic & congenital clinical trials.
This trial is testing a new drug called SRP-1003 for people with myotonic dystrophy type 1 (DM1) who developed symptoms after age 12. The goal is to see if it can reduce muscle problems like myotonia (difficulty relaxing muscles) and improve daily function.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have a confirmed genetic diagnosis of DM1.
- Your symptoms started after age 12 (not congenital DM1).
- You show signs of myotonia (muscles that are slow to relax) as evaluated by a doctor.
- You can walk at least 10 meters (about 33 feet) without help at the start of the study.
- You are not taking medications for myotonia before starting the trial, and you do not have poorly controlled diabetes, high blood pressure, or heart/liver/kidney disease.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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