AVXS-101 Phase 1 gene therapy clinical trial in SMA Type 1: Event free survival and achievement of developmental milestones

Autor: Mendell, Jerry R., Al-Zaidy, Samiah, Shell, Richard, Arnold, W. David, Rodino-Klapac, Louise, Prior, Thomas W., Lowes, Linda, Alfano, Lindsay, Berry, Katherine, Church, Kathleen, Kissel, John T., Nagendran, Sukumar, L'Italien, James, Sproule, Douglas M., Cardenas, Jessica, Burghes, Arthur H.M., Foust, K.D., Meyer, Kathrin, Likhite, Shibi, Kaspar, Brian K.
Zdroj: In European Journal of Paediatric Neurology June 2017 21 Supplement 1:e13-e14
Databáze: ScienceDirect
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