Neuronal ceroid lipofuscinosis, domestic cat
MONDO:1011571Neuronal ceroid lipofuscinosis that occurs in domestic cat.
3 clinical trials for this condition and its sub-types, 0 tagged with Neuronal ceroid lipofuscinosis, domestic cat itself.
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Sub-types of Neuronal ceroid lipofuscinosis, domestic cat
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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