is a rare, maternally-inherited genetic disorder. LHON typically causes vision loss and as a result, affects patients and their families socially, emotionally, and financially. Onset of vision loss due to LHON typically occurs between 15 and 25 years of age, although people outside this age range can also be affected. LHON typically results in severe central vision loss in both eyes, typically one after the other, over an 8-week period. 

Without prior knowledge of a family history, more common causes for the vision loss are often explored before LHON is accurately diagnosed. In most countries, there is no fully approved treatment for LHON.

See if You Qualify


The RESCUE Study is being conducted by GenSight Biologics. To learn more about the company please visit

GenSight Biologics is conducting a clinical trial to evaluate the efficacy of an investigational gene therapy named GS010, which aims to improve vision in patients who have lost vision in the past 6-months due to LHON. The treatment, is a gene therapy given via a standard intravitreal injection into the eye.

You may be eligible if you:

Leber’s Hereditary Optic Neuropathy (LHON)

•Are 15 years of age or over 

•Have the ND4/G11778A gene mutation, without the other primary mutations associated with LHON (ND1/G3460A or ND6/T14484C), as documented by genotyping 

•Have lost vision due to LHON within the past 6 months in at least one of your eyes 

•Meet the other inclusion and exclusion criteria for the clinical trial, as discussed with your doctor and the specialist study team 

The clinical trial will last almost two years, and will involve 12 visits to a specialist clinic. Travel costs and other appropriate expenses for taking part in the trial, including those for one accompanying companion will be supported, in accordance with GenSight Biologics’ internal policies.

If you or a loved one is interested in participating in the RESCUE Study, please fill out the form and a study representative will be in touch. 

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