title: Treatment of fragile X-associated tremor ataxia syndrome (FXTAS) and related neurological problems.
abstract: Fragile X-associated tremor/ataxia syndrome (FXTAS) is a progressive neurological disorder that affects older adult carriers, predominantly males, of premutation alleles (55 to 200 CGG repeats) of the fragile X (FMR1) gene. Principal features of FXTAS are intention tremor, ataxia, parkinsonism, cognitive decline, and peripheral neuropathy; ancillary features include, autonomic dysfunction, and psychiatric symptoms of anxiety, depression, and disinhibition. Although controlled trials have not been carried out in individuals with FXTAS, there is a significant amount of anecdotal information regarding various treatment modalities. Moreover, there exists a great deal of evidence regarding the efficacy of various medications for treatment of other disorders (eg, Alzheimer disease) that have substantial phenotypic overlap with FXTAS. The current review summarizes what is currently known regarding the symptomatic treatment, or potential for treatment, of FXTAS.
authors.affiliation: MIND Institute, University of California, Davis, School of Medicine, Sacramento, CA 95817, USA. randi.hagerman@ucdmc.ucdavis.edu
authors.forename: Randi J
authors.initials: RJ
authors.lastname: Hagerman
authors.forename: Deborah A
authors.initials: DA
authors.lastname: Hall
authors.forename: Sarah
authors.initials: S
authors.lastname: Coffey
authors.forename: Maureen
authors.initials: M
authors.lastname: Leehey
authors.forename: James
authors.initials: J
authors.lastname: Bourgeois
authors.forename: John
authors.initials: J
authors.lastname: Gould
authors.forename: Lin
authors.initials: L
authors.lastname: Zhang
authors.forename: Andreea
authors.initials: A
authors.lastname: Seritan
authors.forename: Elizabeth
authors.initials: E
authors.lastname: Berry-Kravis
authors.forename: John
authors.initials: J
authors.lastname: Olichney
authors.forename: Joshua W
authors.initials: JW
authors.lastname: Miller
authors.forename: Amy L
authors.initials: AL
authors.lastname: Fong
authors.forename: Randall
authors.initials: R
authors.lastname: Carpenter
authors.forename: Cathy
authors.initials: C
authors.lastname: Bodine
authors.forename: Louise W
authors.initials: LW
authors.lastname: Gane
authors.forename: Edgar
authors.initials: E
authors.lastname: Rainin
authors.forename: Hillary
authors.initials: H
authors.lastname: Hagerman
authors.forename: Paul J
authors.initials: PJ
authors.lastname: Hagerman
mesh_terms: D000368:Aged; D000369:Aged, 80 and over; D001259:Ataxia / Q000235:genetics / Q000628:therapy*; D051860:Fragile X Messenger Ribonucleoprotein 1 / Q000235:genetics; D020271:Heredodegenerative Disorders, Nervous System / Q000235:genetics / Q000628:therapy*; D006579:Heterozygote; D006801:Humans; D008297:Male; D008875:Middle Aged; D020734:Parkinsonian Disorders / Q000235:genetics / Q000628:therapy
chemical_list: D051860:Fragile X Messenger Ribonucleoprotein 1