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Therapeutic options and treatment strategies for LHON were discussed by Dr. Valerio Carelli and Dr. Patrick Yu-Wai-Man.  

Therapeutic Options in LHON - Dr. Valerio Carelli

Treatment Strategies for LHON - Hope & Challenges - Dr. Patrick Yu-Wai-Man

In the U.S. and most other countries, there is no approved treatment for LHON. In Europe, a product called Raxone made by Santhera Pharmaceuticals and distributed by Chiesi Pharmaceuticals was approved by the European Medicine Agency (EMA) for use soon after LHON onset and is available in some countries. Raxone is the pharmaceutical grade of idebenone, a synthetic form of CoQ10. The RHODOS clinical trial led to approval.

Idebenone Clinical Trial as Reported in Brain, 2011

There has been much additional research about idebenone, including this:  

Idebenone Retrospective Report in Brain, 2011

Results of another, larger, longer study about idebenone/Raxone called LEROS are expected to publish soon.

It's important to note that LHON experts strongly discourage unaffected LHON carriers from taking idebenone.

Some LHON experts encourage those taking idebenone to avoid taking CoQ10 at the same time, as explained in this video.

Idebenone & CoQ10

LHON experts strongly encourage LHON patients to avoid getting caught up in the hype of for-profit organizations offering “stem cell treatments” to “cure” LHON and other disorders, as they are basically “selling hope to desperation”. There has not been a single scientific, peer-reviewed paper backing up the claims made by these organizations as this type of treatment simply cannot work because:

  1. It’s literally impossible for cells injected either in the blood or CSF (spinal fluid) to get to where they belong in the retina,

  2. There’s no way these cells would know to become Retinal Ganglion Cells (RCG’s), the cells that atrophy in LHON,

  3. Making new RCG’s means nothing if they don’t know where their axons need to connect to.

FDA Warns About Stem Cell Claims

Further, LHON experts warn against other, unproven "treatments."

Treatments to Avoid

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