What are the treatments for Fragile X-Associated Tremor and Ataxia Syndrome (FXTAS)?

Certain medications and therapies are helpful for treating symptoms of FXTAS and may help slow its progression. However, no treatment can stop FXTAS from progressing and none is considered a cure.

Symptoms of FXTAS differ from person to person, and treatment should address a person's individual needs. Healthcare providers may work as a team to provide appropriate treatment, such as physical therapy for difficulty with movement and balance, medication for tremors, and psychological counseling and support services for the individual and family. Other therapies may include rehabilitative treatments such as speech and occupational therapy and gait training. A urologist may be consulted regarding sexual health.1

In addition, the children of FXTAS patients may want to confirm their FMR1 gene status and to discuss their situation with a genetic counselor.

In many cases, medications have been used to treat symptoms associated with FXTAS, as shown in the table below.

Please note that NICHD does not endorse or support the use of any of these medications in treating symptoms of FXTAS, or for other conditions for which the medications are not approved by the U.S. Food and Drug Administration.

Symptoms Medications
Shaking (tremor)
  • Beta blockers
  • Primidone
  • Topiramate
Problems with thinking and memory Most medications for FXTAS-associated cognitive problems and dementia are off-label uses of therapies used for Alzheimer's disease.
Psychiatric symptoms such as anxiety, irritability, agitation, hostility, obsessive-compulsiveness, apathy, and depression
  • Selective serotonin reuptake inhibitors (SSRIs)

Other medications for urinary urgency, bowel incontinence, difficulty swallowing, dizzy spells, high blood pressure, pain, and thyroid dysfunction may also be used.3

Citations

  1. National Fragile X Foundation. (2011). Fragile X-associated tremor/ataxia syndrome (FXTAS). Retrieved May 18, 2012, from https://fragilex.org/understanding-fragile-x/tremor-ataxia-syndrome-fxtas/ external link
  2. Hagerman, R. J., Hall, D. A., Coffey, S., Leehey, M. A., Bourgeois, J., Gould, J., et al. (2008). Treatment of fragile X-associated tremor/ataxia syndrome (FXTAS) and related neurological problems. Clinical Interventions in Aging, 3:251–262.
  3. Leehey, M. A. (2009), Fragile X-associated tremor/ataxia syndrome (FXTAS): clinical phenotype, diagnosis and treatment. Journal of Investigative Medicine, 57, 830–836.
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