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Neuronal Ceroid-Lipofuscinoses
A group of severe neurodegenerative diseases characterized by intracellular accumulation of autofluorescent wax-like lipid materials (CEROID; LIPOFUSCIN) in neurons. There are several subtypes based on mutations of the various genes, time of disease onset, and severity of the neurological defects such as progressive DEMENTIA; SEIZURES; and visual failure.
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Approved for this indication (1)
Phase 2 trials (6)
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Organization Involved with Phase 2 Indications (6)
Organization Involved with Phase 1 Indications (6)