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This search function provides links to outputs produced by NIHR Innovation Observatory. These are briefing notes or reports on new or repurposed technologies. This search will not return all technologies currently in development as these outputs are produced as required for our stakeholders.

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

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

Drugs

Eye Disease

December 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.

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Lenadogene for Vision Loss in Leber's Hereditary Optic Neuropathy



 

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