Gene therapy trial for myotonic dystrophy type 1
Part of Bones, joints & muscles, Brain & nervous system, Genetic & congenital clinical trials.
This trial tests a one-time gene therapy (SAR446268) for people with non-congenital myotonic dystrophy type 1. It aims to see if the treatment is safe and can improve muscle symptoms like stiffness and weakness.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are between 10 and 55 years old (depending on the study part)
- You have non-congenital myotonic dystrophy type 1 (DM1) with muscle stiffness and weakness confirmed by a doctor
- You have a genetic test showing a CTG repeat length of 50 or more
- You can walk at least 10 meters on your own (using braces or orthotics is okay)
- You have not had prior treatment that would interfere with the gene therapy
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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