Personalized treatment for GARS1-related CMT2D
Part of Brain & nervous system, Genetic & congenital clinical trials.
This trial tests a personalized medicine designed for a single person with a specific genetic mutation (GARS1) that causes Charcot-Marie-Tooth disease type 2D. The treatment is an antisense oligonucleotide, a custom-made molecule that targets the faulty gene.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have a confirmed mutation in the GARS1 gene.
- You must be able to travel to the study location and follow all study visits.
- You must be willing to provide your medical records and give consent (or have a parent/guardian provide it).
- You cannot have any other health condition that, in the study doctor's opinion, would prevent you from completing 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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