P.350Onasemnogene abeparvovec gene-replacement therapy (GRT) in pre-symptomatic spinal muscular atrophy (SMA)
| Autor: | Schultz, M., Swoboda, K., Farrar, M., McMillan, H., Parsons, J., Kernbauer, E., Farrow, M., Ogrinc, F., Kavanagh, S., Feltner, D., McGill, B., Spector, S., L'Italien, J., Sproule, D., Strauss, K. |
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| Zdroj: | In Neuromuscular Disorders October 2019 29 Supplement 1:S183-S183 |
| Databáze: | ScienceDirect |
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