Gene therapy for CLN6 batten disease
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This trial tests a gene therapy that may help treat CLN6 Batten disease, a rare inherited condition that affects the nervous system. The therapy aims to replace the faulty gene to slow or stop the disease from getting worse.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have a confirmed diagnosis of CLN6 Batten disease.
- You must be at least 4 months old.
- You cannot have another inherited brain or nerve disease.
- You cannot have had a stem cell transplant before.
- You cannot have had gene therapy, gene editing, or viral vector treatment 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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