Treatments to Consider and Those to Avoid
Therapeutic Options Overview
Therapeutic options and treatment strategies for LHON were discussed in the following videos by Dr. Patrick Yu-Wai-Man and Dr. Valerio Carelli.
In the U.S. and most other countries, there is no approved treatment for LHON. A product called Raxone 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 following are some of the many articles regarding idebenone and LHON.
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 the following Idebenone & CoQ10 video.
Clinical Trials
Once diagnosed, it’s good to discuss with your medical professional if there are any legitimate clinical trials underway for which you may be eligible, or if taking specific supplements would be appropriate for you.
The clinical trials.gov website is a good resource. Note that being listed on the site is not an endorsement of the study’s safety and science.
This map provides information about clinical trials being conducted in the EU.
LHON Medical Registry
To help further research that may lead to the development of treatments for LHON, participate in our LHON global medical registry at RARE-X.org. This initiative is for unaffected or affected carriers of an LHON mutation, because both groups have the genetic mutation, and your information will add to the research possibilities for LHON. More information on this topic can be found on the Medical Registry page of our website.
Treatments to Avoid
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:
2. There’s no way these cells would know to become Retinal Ganglion Cells (RGC’s), the cells that atrophy in LHON.
3. Making new RGC’s means nothing if they don’t know where their axons need to connect.
LHON experts warn against other, unproven “treatments” in the following Treatments to Avoid video.
