Home Diseases and Conditions Leigh Syndrome

Leigh Syndrome

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Development Stage

Early Stage

Focused ultrasound research is in the laboratory phase and is not yet available for patients.

Clinical Trials

Focused ultrasound for this condition is being researched in clinical trials.

International Approval

Focused ultrasound is approved to treat this condition outside the US. Patients can seek commercial treatment at participating international sites.

FDA Approved

The US Food and Drug Administration has approved focus ultrasound for this condition. Patients can seek commercial treatment at participating sites.

Early Stage

Clinical Trials

International

FDA Approved

Focused ultrasound research for this condition is in the laboratory phase and is not yet available for patients.

Focused Ultrasound Therapy 

Focused ultrasound is a therapeutic technology that could transform the quality of life and decrease the cost of care for patients with Leigh syndrome This novel technology focuses beams of ultrasound energy precisely and accurately on targets deep in the body without damaging surrounding normal tissue. 

How it Works 
Where the beams converge, focused ultrasound produces a therapeutic effect where the target area blood-brain barrier (BBB) is temporarily opened, and therapeutic agents in the blood stream have the opportunity to get access to the brain and improve the neurodegeneration that has been occurring.  

For certain patients, focused ultrasound could provide a noninvasive and more effective alternative to existing care. While significant work has been accomplished, there is still much to do be done before this technology will be widely available.

Advantages 

  • Focused ultrasound is noninvasive, 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 Leigh syndrome.  

The Foundation updates these pages regularly, but with the increasing number of clinical trials, we want to be sure that our audience has the latest information available. Therefore, we also added the website search information for the above trials. If you click here, it will take you to the latest information available from https://www.clinicaltrials.gov/.  

Regulatory Approval and Reimbursement  

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

Notable Papers 

Faideau M, Clément R, Rigollet S, Benegiamo G, Cresson C, Blot B, Reynaud-Dulaurier R, Yjjou S, Aprahamian F, Durand S, Delalande A, Barbier EL, Stupar V, Auwerx J, Decressac M. Ultrasound-assisted gene therapy mitigates Leigh syndrome pathology. Brain. 2026 Jan 23:awag026. doi: 10.1093/brain/awag026. Epub ahead of print. PMID: 41572892. 

Click here for additional references from PubMed.