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Intravitreal Gene Therapy vs. Natural History in Patients With Leber Hereditary Optic Neuropathy Carrying the m.11778G>A ND4 Mutation: Systematic Review and Indirect Comparison
Objective: This work aimed to compare the evolution of visual outcomes in Leber hereditary optic neuropathy (LHON) patients treated with intravitreal gene therapy to the spontaneous evolution in prior natural history (NH) studies. Design: A combined analysis of two phase three randomized, double-mas...
Autores principales: | Newman, Nancy J., Yu-Wai-Man, Patrick, Carelli, Valerio, Biousse, Valerie, Moster, Mark L., Vignal-Clermont, Catherine, Sergott, Robert C., Klopstock, Thomas, Sadun, Alfredo A., 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 |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Frontiers Media S.A.
2021
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8181419/ https://www.ncbi.nlm.nih.gov/pubmed/34108929 http://dx.doi.org/10.3389/fneur.2021.662838 |
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