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

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Therapeutic Areas: Genetic Disorders , Ophthalmology
Year: 2017

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.