Focused Ultrasound Therapy

arly Stage squareFocused ultrasound is an early-stage, non-invasive, therapeutic technology with the potential to improve the quality of life and decrease the cost of care for patients with Rett Syndrome. This novel technology focuses beams of ultrasound energy precisely and accurately on targets deep in the brain without damaging surrounding normal tissue. Where the beams converge, there are a couple of methods of action. In one approach the ultrasound may facilitate the introduction of therapeutic genetic material. The other approach is to produce enhanced activity of Brain Derived Neurotrophic Factor (BDNF) which could reduce the impact for patients with Rett Syndrome. While significant preclinical work has been accomplished, there is still much to be done before either technology will be widely available.

Existing therapy for Rett Syndrome is supportive and symptomatic.

Focused ultrasound could provide a non-invasive therapy for Rett Syndrome.


  • Focused ultrasound is non-invasive, so it does not carry added concerns like surgical wound healing or infection.
  • Focused ultrasound can reach the desired target without damaging surrounding tissue.
  • It can be repeated, if necessary.

Clinical Trials

At the present time, there are no clinical trials recruiting patients for focused ultrasound treatment of Rett Syndrome.

Regulatory Approval and Reimbursement 

Focused ultrasound treatment for Rett Syndrome is not yet approved by regulatory bodies or covered by medical insurance companies.

See here for a list of laboratory research sites.

Notable Papers

Matagne V, Ehinger Y, Saidi L, Borges-Correia A, Barkats M, Bartoli M, Villard L, Roux JC. A codon-optimized Mecp2 transgene corrects breathing deficits and improves survival in a mouse model of Rett syndrome. Neurobiol Dis. 2017 Mar;99:1-11. doi: 10.1016/j.nbd.2016.12.009. Epub 2016 Dec 11.

Tsai, SJ. Therapeutic Potential of Transcranial Focused Ultrasound for Rett Syndrome. Med Sci Monit. 2016 Oct 27;22:4026-4029. PMID: 27786169, PMCID: PMC5087669, DOI: 10.12659/msm.898041

Click here for additional references from PubMed.


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