Long-Term Follow-Up After Unilateral Intravitreal Gene Therapy for Leber Hereditary Optic Neuropathy: The RESTORE Study

RESCUE and REVERSE were 2 Phase 3 clinical trials that assessed the efficacy and safety of intravitreal gene therapy with lenadogene nolparvovec (rAAV2/2-ND4) for the treatment of Leber hereditary optic neuropathy (LHON). RESTORE is the long-term follow-up study of subjects treated in the RESCUE and...

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Detalles Bibliográficos
Autores principales: Biousse, Valérie, Newman, Nancy J., Yu-Wai-Man, Patrick, Carelli, Valerio, Moster, Mark L., Vignal-Clermont, Catherine, Klopstock, Thomas, Sadun, Alfredo A., Sergott, Robert C., Hage, Rabih, Esposti, Simona, La Morgia, Chiara, Priglinger, Claudia, Karanja, Rustum, Blouin, Laure, Taiel, Magali, Sahel, José-Alain
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Journal of Neuro-Ophthalmology 2021
Materias:
Original Contribution
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8366761/
https://www.ncbi.nlm.nih.gov/pubmed/34415265
http://dx.doi.org/10.1097/WNO.0000000000001367

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