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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Broader categories
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Experimental gene therapy aims to halt rare childhood brain disease
Disease control Not yet recruitingThis early-phase trial tests a gene therapy for children with CLN6 Batten disease, a rare and fatal brain disorder. Twelve children will receive a single injection of the therapy into the fluid around the spinal cord. Researchers will check if it is safe and if it can slow the di…
Phase: PHASE1, PHASE2 • Sponsor: The Charlotte and Gwenyth Gray Foundation • Aim: Disease control
Last updated Jun 27, 2026 12:34 UTC
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Could a common cholesterol drug slow batten disease?
Disease control Not yet recruitingThis phase 3 trial tests whether PLX-200 (gemfibrozil) can safely slow the progression of CLN3 disease, a rare genetic disorder that causes loss of motor skills, vision, and seizures in children. About 39 kids aged 6-18 will receive either the drug or a placebo for up to 96 weeks…
Phase: PHASE3 • Sponsor: Polaryx Therapeutics, Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:30 UTC