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Study design and baseline characteristics for the reflect gene therapy trial ofm.11778g>A/ND4-LHON

OBJECTIVE: REFLECT is the first randomised, double-masked, placebo-controlled multicentre phase 3 clinical trial that evaluated the efficacy and safety of bilateral intravitreal (IVT) injection of lenadogene nolparvovec in subjects with Leber hereditary optic neuropathy carrying the m.11778G>A mu...

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Detalles Bibliográficos
Autores principales: Subramanian, Prem S, Newman, Nancy J, Moster, Mark, Wang, An-Guor, Yu-Wai-Man, Patrick, Donahue, Sean, Leroy, Bart P, Carelli, Valerio, Biousse, Valerie, Vignal-Clermont, Catherine, Sergott, Robert C, Sadun, Alfredo A, Rebolleda, Gema, Chwalisz, Bart K, Banik, Rudrani, Bazin, Fabienne, Cox, Eric, Roux, Michel, Taiel, Magali, Sahel, Jose-Alain
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BMJ Publishing Group 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9723886/
http://dx.doi.org/10.1136/bmjophth-2022-001158