BATTEN DISEASE
Clinical trials for BATTEN DISEASE explained in plain language.
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Experimental gene therapy aims to halt rare fatal brain disease in children
Disease control OngoingThis early-stage trial tests a one-time gene therapy called CLN-301 in 7 children aged 3 to 10 with CLN3 Batten disease, a rare genetic disorder that causes progressive loss of vision, movement, and thinking skills. The therapy delivers a working copy of the CLN3 gene directly in…
Matched conditions: BATTEN DISEASE
Phase: PHASE1, PHASE2 • Sponsor: Neela Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 13:06 UTC
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Gene therapy for rare brain disease tracked over years
Disease control OngoingThis study follows 10 people with CLN6 Batten disease who received a single dose of gene therapy (AT-GTX-501) in an earlier trial. Researchers are checking long-term safety and how the disease progresses over time. No new treatment is given in this follow-up.
Matched conditions: BATTEN DISEASE
Sponsor: Emily de los Reyes • Aim: Disease control
Last updated Jun 27, 2026 07:52 UTC
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New study aims to measure how CLN2 disease affects Children's development
Knowledge-focused ENROLLING_BY_INVITATIONThis study looks at how children with CLN2 Batten disease develop over time, including their thinking, language, and motor skills. Researchers will compare children who receive treatment (cerliponase alfa) with those who do not. The goal is to find better ways to measure the dise…
Matched conditions: BATTEN DISEASE
Sponsor: Jessica Scherr • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:55 UTC