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

<p>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&thinsp;months of follow-up in adult males with choroideremia randomized to receive a high-dose (1.0&thi...

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書誌詳細
主要な著者: MacLaren, RE, Fischer, MD, Gow, JA, Lam, BL, Sankila, E-MK, Girach, A, Panda, S, Yoon, D, Zhao, G, Pennesi, ME
フォーマット: Journal article
言語:English
出版事項: Springer Nature 2023

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