Article
Safety of Intravitreal Gene Therapy for Treatment of Subjects with Leber Hereditary Optic Neuropathy due to Mutations in the Mitochondrial ND4 Gene: The REVEAL Study.
BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy - 1 Mar 2021
Vignal-Clermont Catherine, Girmens Jean-François, Audo Isabelle, Said Saddek Mohand, Errera Marie-Hélène, Plaine Lise, O'Shaughnessy Denis, Taiel Magali, Sahel José-Alain
Abstract excerpt
BACKGROUND: Leber hereditary optic neuropathy (LHON) is a maternally inherited mitochondrial disease whose primary clinical manifestation is bilateral visual loss. Only a single therapy, idebenone, is approved in Europe for use in exceptional circumstances and no therapy is currently approved in the USA. LHON remains a disease with a high unmet medical need. OBJECTIVE: This is a report of an open-label,...
Topics
- Europe
- Genetic Therapy
- Humans
- Mutation
- Optic Atrophy, Hereditary, Leber
- Tomography, Optical Coherence
- Visual Fields