Gene-replacement therapy (GRT) in spinal muscular atrophy type 1 (SMA1): Long-term follow-up from the onasemnogene abeparvovec phase 1/2A clinical trial
| Autor: | Mendell, J.R., Shell, R., Lehman, K.J., McColly, M., Lowes, L.P., Alfano, L.N., Miller, N.F., Iammarino, M.A., Church, K., Spector, S.A., Ogrinc, F.G., Ouyang, H., Kernbauer, E., Shah, S., L’Italien, J., Sproule, D.M., Feltner, D.E., Al-Zaidy, S. |
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| Zdroj: | In Journal of the Neurological Sciences 15 October 2019 405 Supplement:271-271 |
| Databáze: | ScienceDirect |
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