First CRISPR RNA therapy for MECP2 duplication syndrome
Part of Brain & nervous system, Genetic & congenital clinical trials.
This trial tests a new CRISPR therapy that edits RNA to treat a rare genetic condition called MECP2 duplication syndrome (MDS). It is for boys aged 2 to 18 who have stable seizures and no other major health problems, with the goal of improving symptoms and safety.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Boys between ages 2 and 18 at the time of signing up
- Must have a confirmed diagnosis of MECP2 duplication syndrome (MDS) through genetic testing
- Seizures, if present, must be stable for at least 2 months before starting the trial
- Will need to stay in the hospital for a special injection into the fluid around the brain and give blood and urine samples
- Cannot have other serious health issues like significant brain damage, heart disease, or have had gene therapy before
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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