Study of Batten disease progress and clinical changes over time
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This trial tracks how Batten disease (NCL) affects the body and how symptoms change over time. If you have a confirmed genetic (molecular) diagnosis—especially CLN2 with Brineura treatment—it may help researchers understand disease patterns and care needs.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have a confirmed genetic (molecular) diagnosis of NCL/Batten disease
- The diagnosis must be documented, not just suspected
- If you’re in the CLN2 group: you have documented TPP1 deficiency
- If you’re in the CLN2 group: you have had treatment with Brineura (cerliponase alpha) into the brain’s fluid spaces
- You may still join if you’re in other post-marketing studies
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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