Lenadogene Nolparvovec (GS-010) for Vision Loss from Leber’s Hereditary Optic Neuropathy Due to Mutation of the ND4 Gene

There are currently very few treatment options for LHON. Most treatment methods are used to support the patient rather than cure them. Lenadogene nolparvovec is a new gene-targeted treatment intended for vision loss caused by LHON due to an alteration in a specific gene. As a genetic medicine, lenadogene nolparvovec could be a promising option to treat LHON and if licensed, could provide a new treatment option for those with LHON.