Gene therapy for CLN6 Batten disease
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This study tests a gene therapy treatment for CLN6 Batten disease, a rare inherited condition that affects the brain and nervous system. The therapy aims to slow or stop disease progression by introducing a healthy copy of the faulty gene.
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 neurologic disease (separate from CLN6)
- You cannot have had a stem cell transplant in the past
- You cannot have received prior gene therapy, gene editing, or viral vector treatments
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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