Study medicine for DM1 to improve muscle function
Part of Bones, joints & muscles, Brain & nervous system, Genetic & congenital clinical trials.
This early-stage study tests DYNE-101 to see how safe it is and whether it can improve measurable muscle function in people with myotonic dystrophy type 1 (DM1). You may be asked to do several walking and strength tests and undergo heart and breathing screening measurements.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have a confirmed DM1 diagnosis with a DM1 gene repeat count over 100 (your genetic test result).
- Your first DM1 muscle symptoms started at age 12 or older.
- You have noticeable muscle stiffness (“myotonia”)—for example, your hand takes at least 2 seconds to open after being gripped (as judged at screening).
- You can complete screening strength and movement tests without help (no cane/walker; no braces/orthoses for the tests): 10-meter walk, stair climb (in some groups), and 5-times sit-to-stand.
- Your recent medical history doesn’t include major surgery in the last 12 weeks (or plans for major surgery during the study).
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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