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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...
| Autores principales: | , , , , , , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
Nature Publishing Group US
2023
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| 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 |