Juvenile neuronal ceroid lipofuscinosis
MONDO:0019262A genetically heterogeneous group of neuronal ceroid lipofuscinoses (NCLs) typically characterized by onset at early school age with vision loss due to retinopathy, seizures and the decline of mental and motor capacities.
Also known as: JNCL, Spielmeyer-Vogt disease, batten disease, juvenile NCL, juvenile neuronal ceroid lipofuscinosis
6 clinical trials for this condition and its sub-types.
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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…
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.
Sponsor: Emily de los Reyes • Aim: Disease control
Last updated Jun 27, 2026 07:52 UTC