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Subretinal timrepigene emparvovec in adult men with choroideremia: a randomized phase 3 trial

Choroideremia is a rare, X-linked retinal degeneration resulting in progressive vision loss. A randomized, masked, phase 3 clinical trial evaluated the safety and efficacy over 12 months of follow-up in adult males with choroideremia randomized to receive a high-dose (1.0 × 10(11) vector genomes (vg...

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Detalles Bibliográficos
Autores principales: MacLaren, Robert E., Fischer, M. Dominik, Gow, James A., Lam, Byron L., Sankila, Eeva-Marja K., Girach, Aniz, Panda, Sushil, Yoon, Dan, Zhao, Guolin, Pennesi, Mark E.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group US 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10579095/
https://www.ncbi.nlm.nih.gov/pubmed/37814062
http://dx.doi.org/10.1038/s41591-023-02520-3