disease 1,138 words KG: ent-dise-4f58f196 2026-03-19
kind:diseasesection:diseasesstate:published
Contents

Fragile X-Associated Tremor/Ataxia Syndrome (FXTAS)

Disease Info
PrevalenceFXTAS affects approximately 1 in 450-500 males and 1 in 150-300 females who carry the *FMR1* premutation[@tassone2012]
Typical OnsetTypically begins in the sixth decade of life (50s-60s)
PenetranceFull penetrance by the eighth decade in males; females typically have milder symptoms due to X-inactivation
Sex DistributionMore common and severe in males, reflecting the single X chromosome in males
Normal< 45 CGG repeats
Premutation55-200 CGG repeats
Full Mutation> 200 CGG repeats (causes Fragile X syndrome)
FMR1 (Fragile X Mental Retardation 1)The causative gene; located on chromosome Xq27.3
Brain AtrophyCerebellar atrophy (especially of the cerebellar vermis) and cerebral white matter changes
Intranuclear InclusionsUbiquitin-positive, FMRpolyG-containing inclusions in neurons and astrocytes throughout the brain[@hagerman2020]
Loss of Purkinje CellsDegeneration of cerebellar Purkinje cells contributes to ataxia
White Matter HyperintensitiesMRI shows T2/FLAIR hyperintensities in the middle cerebellar peduncles (MCP signs) and periventricular white matter
DatabasesOMIMOrphanetClinicalTrialsPubMed

Knowledge Graph