PGN-EDODM1 for myotonic dystrophy type 1
Part of Bones, joints & muscles, Brain & nervous system, Genetic & congenital clinical trials.
This study tests an experimental drug called PGN-EDODM1 for people with myotonic dystrophy type 1 (DM1). The goal is to see if it can help reduce symptoms like muscle stiffness (myotonia).
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have a confirmed diagnosis of myotonic dystrophy type 1 (DM1) with at least 100 CTG repeats in the DMPK gene.
- You must have myotonia, which is muscle stiffness or trouble relaxing muscles after using them.
- You must have enough muscle in your lower legs (tibialis anterior) for a safe needle biopsy.
- Your body mass index (BMI) must be below 35.
- You cannot have the congenital (from birth) form of DM1.
- You cannot have taken any medication specifically for myotonia within the last 2 weeks.
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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