The clinical and pathological features of two unrelated sporadic cases of neuroaxonal dystrophy occurring in young adults are described. Initial clinical manifestations in both patients were those of primary psychiatric disorders. They subsequently developed extrapyramidal symptoms, dementia, cerebellar ataxia, and corticospinal dysfunction, but neither demonstrated myoclonic seizures. In one patient the diagnosis was made before death by brain biopsy. On pathological examination both patients showed a generalized distribution of spheroids within the central nervous system and, in one, in the peripheral nerves. In both patients, Lewy bodies were demonstrated in the pigmented brainstem nuclei. In addition, one patient showed ultrastructurally verified Lewy bodies in the cerebral cortex. Although these two cases could be considered examples of juvenile neuroaxonal dystrophy, we prefer to regard them as transitional forms in a disease spectrum with classic infantile neuroaxonal dystrophy and Hallervorden-Spatz disease at the two extremes.
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http://dx.doi.org/10.1002/ana.410110403 | DOI Listing |
Int J Mol Sci
January 2025
Department of Human Genetics, School of Public Health, University of Pittsburgh, Pittsburgh, PA 15261, USA.
A couple presented to the office with an apparently healthy infant for a thorough clinical assessment, as they had previously lost two male children to a neurodegenerative disorder. They also reported the death of a male cousin abroad with a comparable condition. We aimed to evaluate a novel coding pathogenic variant c.
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Neuroscience Group of Antioquia (GNA) and Molecular Genetics Group (GENMOL), University of Antioquia, Medellín 050010, Colombia.
Neurogenetics
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Department of Medical Genetics, Shahid Beheshti University of Medical Sciences, Tehran, Iran.
Orphanet J Rare Dis
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Andalusian Centre for Developmental Biology-CSIC-Pablo de Olavide University, 41013, Seville, Spain.
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