SMA THERAPIES I: P.180AVXS-101 phase 1 gene replacement therapy clinical trial in spinal muscular atrophy type 1: patients treated early with the proposed therapeutic dose were able to sit unassisted at a younger age
| Autor: | Alfano, L., Lowes, L., Al-Zaidy, S., Shell, R., Arnold, W., Rodino-Klapac, L., Prior, T., Berry, K., Church, K., Kissel, J., Nagendran, S., Italien, J.L., Sproule, D., Wells, C., Burghes, A., Foust, K., Meyer, K., Likhite, S., Kaspar, B., Mendell, J. |
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| Zdroj: | In Neuromuscular Disorders October 2018 28 Supplement 2:S82-S82 |
| Databáze: | ScienceDirect |
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