Fragile X-associated tremor/ataxia syndrome: clinical features, genetics, and testing guidelines

Details

Serval ID
serval:BIB_DD7787BAFFE1
Type
Article: article from journal or magazin.
Publication sub-type
Review (review): journal as complete as possible of one specific subject, written based on exhaustive analyses from published work.
Collection
Publications
Title
Fragile X-associated tremor/ataxia syndrome: clinical features, genetics, and testing guidelines
Journal
Movement Disorders
Author(s)
Berry-Kravis  E., Abrams  L., Coffey  S. M., Hall  D. A., Greco  C., Gane  L. W., Grigsby  J., Bourgeois  J. A., Finucane  B., Jacquemont  S., Brunberg  J. A., Zhang  L., Lin  J., Tassone  F., Hagerman  P. J., Hagerman  R. J., Leehey  M. A.
ISSN
0885-3185
Publication state
Published
Issued date
10/2007
Peer-reviewed
Oui
Volume
22
Number
14
Pages
2018-30, quiz 2140
Notes
Journal Article
Review --- Old month value: Oct 31
Abstract
Fragile X-associated tremor/ataxia syndrome (FXTAS) is a neurodegenerative disorder with core features of action tremor and cerebellar gait ataxia. Frequent associated findings include parkinsonism, executive function deficits and dementia, neuropathy, and dysautonomia. Magnetic Resonance Imaging studies in FXTAS demonstrate increased T2 signal intensity in the middle cerebellar peduncles (MCP sign) in the majority of patients. Similar signal alterations are seen in deep and subependymal cerebral white matter, as is general cortical and subcortical atrophy. The major neuropathological feature of FXTAS is the presence of intranuclear, neuronal, and astrocytic, inclusions in broad distribution throughout the brain and brainstem. FXTAS is caused by moderate expansions (55-200 repeats; premutation range) of a CGG trinucleotide in the fragile X mental retardation 1 (FMR1) gene, the same gene which causes fragile X syndrome when in the full mutation range (200 or greater CGG repeats). The pathogenic mechanism is related to overexpression and toxicity of the FMR1 mRNA per se. Although only recently discovered, and hence currently under-diagnosed, FXTAS is likely to be one of the most common single-gene disorders leading to neurodegeneration in males. In this report, we review information available on the clinical, radiological, and pathological features, and prevalence and management of FXTAS. We also provide guidelines for the practitioner to assist with identifying appropriate patients for DNA testing for FXTAS, as well as recommendations for genetic counseling once a diagnosis of FXTAS is made.
Keywords
Ataxia/*genetics/pathology/physiopathology Family Health Female Fragile X Syndrome/*genetics/pathology/physiopathology Genetic Screening Guidelines as Topic/*standards Humans Magnetic Resonance Imaging/methods Male Sex Factors Tremor/*genetics/pathology/physiopathology
Pubmed
Web of science
Create date
28/02/2008 11:42
Last modification date
20/08/2019 17:02
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