Late infantile neuronal ceroid lipofuscinosis
MONDO:0015674A genetically heterogeneous group of neuronal ceroid lipofuscinoses (NCLs) typically characterized by onset during infancy or early childhood with decline of mental and motor capacities, epilepsy, and vision loss through retinal degeneration.
Also known as: Jansky-Bielschowsky disease, LINCL, late infantile NCL, late-infantile neuronal ceroid lipofuscinosis, Bielschowsky-jansky disease, Bielschowsky-jansky type neuronal ceroid lipofuscinosis, amaurotic idiocy early juvenile type, amaurotic idiocy late infantile type
5 clinical trials for this condition and its sub-types.
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Broader categories
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One-Time gene injection aims to slow rare fatal brain disease in kids
Disease control OngoingThis early-stage study tests a one-time gene therapy injection into the spinal fluid of children aged 1-18 with CLN7 Batten disease, a rare and fatal genetic brain disorder. The main goal is to check safety, but researchers will also measure changes in movement, thinking, and vis…
Phase 1 • Sponsor: Benjamin Greenberg • Aim: Disease control
Last updated Jun 27, 2026 08:07 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