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  • 1.  Clinical trials for LHON in the US

    Posted 09-11-2025 22:03

    Hi,

    This is so unusual – I have seen 3 men in the past week who I think have LHON (and my denominator is small). Genetic testing was ordered on all of them.

    I looked on clincaltrials.gov and did not see any trials that are currently recruiting in the U.S.

    If you are aware of any clinical trials currently looking for participants, please contact me privately.

    Also, if you can recommend a reputable source for idebenone, I would like to pass the information on to my patients.

    Thank you!

    Deb

    DeborahFriedman@tx.rr.com

     



  • 2.  RE: Clinical trials for LHON in the US

    Posted 09-12-2025 16:24
    Yep.  No trials at the moment and the trials in the pipeline are being prepared very slowly.  Just Google Idebenone and LHON.  Most of my patients end up using HBC or Relentless (for the record, they buy it directly; we don't prescribe it).

    Alfredo





  • 3.  RE: Clinical trials for LHON in the US

    Posted 09-12-2025 16:53

    Bummer about the trials. Thanks, Alfredo!

    Deb






  • 4.  RE: Clinical trials for LHON in the US

    Posted 09-12-2025 20:18





  • 5.  RE: Clinical trials for LHON in the US

    Posted 09-19-2025 11:54
    Along the same lines:
    Are there antibiotics you ask carrier if the mutation to avoid- other than Ethambutol and Linezolid.

    Thanks!

    Sangeeta 





  • 6.  RE: Clinical trials for LHON in the US

    Posted 09-12-2025 16:56
    Chemistry Rx is a compounding pharmacy that has idebenone they can make into capsules of different doses. They also have staff that will run prior auths through insurance. I am in the process of doing this for the first time so not sure how it will turn out but it’s the only pharmacy that has offered this.

    Bobby Goodrich MD
    Wake Forest




  • 7.  RE: Clinical trials for LHON in the US

    Posted 09-12-2025 20:17