A group of inherited progressive degenerative brain diseases characterized clinically by a decline of mental and other capacities, epilepsy, and vision loss through retinal degeneration, and histopath...
Comprehensive, easy-to-understand information about this condition
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Organizations with approved therapies for this disease
No approved therapies yet
Research is ongoing — 3 companies have orphan drug designations for this disease
Kisho delivers this disease record via API, including phenotypes (HPO), genes, orphan drug designations, screening status, and PAG mapping, with version history and governance.
AI-curated news mentioning neuronal ceroid lipofuscinosis
Updated Feb 6, 2026
Despite ongoing research, no disease-modifying therapies exist for neuronal ceroid lipofuscinosis, with 5 completed clinical trials and 19 case reports since 1977. The lack of proven treatments highlights the challenges faced in developing effective therapies for this rare disease.