Document Detail


Normomorphic sialidosis in two female adults with severe neurologic disease and without sialyl oligosacchariduria.
MedLine Citation:
PMID:  3096875     Owner:  NLM     Status:  MEDLINE    
Abstract/OtherAbstract:
Two female patients of German origin, aged 38 and 21 years, with myoclonus epilepsy and cerebellar ataxia, but without dysmorphic signs and dementia, were found to excrete normal amounts of sialyl oligosaccharides in their urine. The younger patient showed cherry red spots in her ocular fundi. The older patient had a brother with an autopsy-proven neuronal storage disease compatible with sialidosis, and in her rectal biopsy lamellar inclusion bodies were detected. Enzyme assays in cultured fibroblasts of both patients revealed a profound but incomplete deficiency of oligosaccharide sialidase activity and normal beta-galactosidase activity. Adult sialidosis was diagnosed in both patients. In their fibroblasts, moderate elevations of bound sialic acid could also be measured. The small residual sialidase activity, which in the older patient had a normal KM value, is considered responsible for the late onset and slow clinical course of the disease. It is concluded that in adult sialidosis the extraneural storage process can be difficult to demonstrate in terms of metabolite accumulation or excretion during the course of intraneuronal storage.
Authors:
K Harzer; M Cantz; A C Sewell; S S Dhareshwar; W Roggendorf; R W Heckl; O Schofer; R Thumler; J Peiffer; W Schlote
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Publication Detail:
Type:  Case Reports; Journal Article    
Journal Detail:
Title:  Human genetics     Volume:  74     ISSN:  0340-6717     ISO Abbreviation:  Hum. Genet.     Publication Date:  1986 Nov 
Date Detail:
Created Date:  1987-01-20     Completed Date:  1987-01-20     Revised Date:  2004-11-17    
Medline Journal Info:
Nlm Unique ID:  7613873     Medline TA:  Hum Genet     Country:  GERMANY, WEST    
Other Details:
Languages:  eng     Pagination:  209-14     Citation Subset:  IM    
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MeSH Terms
Descriptor/Qualifier:
Adult
Cells, Cultured
Cerebellar Ataxia / enzymology,  genetics*,  urine
Epilepsies, Myoclonic / enzymology,  genetics*,  urine
Female
Fibroblasts / analysis
Humans
Intestinal Mucosa / pathology
Macula Lutea / pathology
Male
Middle Aged
N-Acetylneuraminic Acid
Neuraminidase / deficiency*
Neurons / pathology
Oligosaccharides / urine
Sialic Acids / urine
Chemical
Reg. No./Substance:
0/Oligosaccharides; 0/Sialic Acids; 131-48-6/N-Acetylneuraminic Acid; EC 3.2.1.18/Neuraminidase

From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine


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