Abstract
The authors describe two Japanese siblings with autosomal recessive spastic ataxia of Charlevoix-Saguenay (ARSACS) without spasticity, usually a core feature of this disorder. They had a novel homozygous missense mutation (T987C) of the SACS gene, which resulted in a phenylalanine-to-serine substitution at amino acid residue 304.
Publication types
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Case Reports
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Research Support, Non-U.S. Gov't
MeSH terms
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Adult
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Afferent Pathways / pathology
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Afferent Pathways / physiopathology
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Age of Onset
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Amino Acid Substitution / genetics
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Ataxia / genetics
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Ataxia / physiopathology*
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Cerebellar Diseases / genetics
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Cerebellar Diseases / pathology
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Cerebellar Diseases / physiopathology*
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Cerebellum / pathology
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Cerebellum / physiopathology
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Chromosome Disorders / genetics
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Chromosome Disorders / physiopathology*
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DNA Mutational Analysis
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Genes, Recessive / genetics*
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Genetic Testing
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Heat-Shock Proteins / genetics*
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Humans
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Japan
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Male
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Muscle Spasticity / genetics
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Muscle Spasticity / physiopathology
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Mutation, Missense / genetics*
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Peripheral Nerves / pathology
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Peripheral Nerves / physiopathology
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Phenotype
Substances
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Heat-Shock Proteins
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SACS protein, human