Indirect Comparison of Lenadogene Nolparvovec Gene Therapy Versus Natural History in Patients with Leber Hereditary Optic Neuropathy Carrying the m.11778G>A MT-ND4 Mutation

INTRODUCTION: Lenadogene nolparvovec is a promising novel gene therapy for patients with Leber hereditary optic neuropathy (LHON) carrying the m.11778G>A ND4 mutation (MT-ND4). A previous pooled analysis of phase 3 studies showed an improvement in visual acuity of patients injected with lenadogen...

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Detalles Bibliográficos
Autores principales: Carelli, Valerio, Newman, Nancy J., Yu-Wai-Man, Patrick, Biousse, Valerie, Moster, Mark L., Subramanian, Prem S., Vignal-Clermont, Catherine, Wang, An-Guor, Donahue, Sean P., Leroy, Bart P., Sergott, Robert C., Klopstock, Thomas, Sadun, Alfredo A., Rebolleda Fernández, Gema, Chwalisz, Bart K., Banik, Rudrani, Girmens, Jean François, La Morgia, Chiara, DeBusk, Adam A., Jurkute, Neringa, Priglinger, Claudia, Karanjia, Rustum, Josse, Constant, Salzmann, Julie, Montestruc, François, Roux, Michel, Taiel, Magali, Sahel, José-Alain
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer Healthcare 2022
Materias:
Original Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9834474/
https://www.ncbi.nlm.nih.gov/pubmed/36449262
http://dx.doi.org/10.1007/s40123-022-00611-x

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