There are many research centres all over the world where LHON is being studied, including clinical trials. Even though there are few people affected by LHON, many researchers are investigating the disease because, as a mitochondrial disorder, it is similar in nature to diseases such as Alzheimer, Huntington, and Parkinson, which affect a great number of patients around the world.
Amongst current research there are:
– Gene therapy – GenSight: Many places around the world [Recruiting at the moment those over 15 years old that are affected by 11778 mutation and have had the disease onset in less than a year.]
– Biologics GenSight – REFLECT Trial – Santhera Pharmaceuticals: In many places in the USA and Europe [Recruiting at the moment those over 12 years old, with confirmed 11778 mutation, 14484 mutation, or 3460 mutation, whose disease onset happened 5 years prior or less, and that never ingested idebenone.]
LEROS trial
Webinar about LEROS trial
Gene therapy – Bascom Palmer, the University of Miami [Recruiting at the moment only those over 15 years old that are affected by 11778 mutation.]: Low and medium doses results.
Stealth BioTherapeutics – Elamipretide [Fully recruited; Data is being collected: Stealth BioTherapeutics initiated a longitudinal study about a family affected by LHON.]