SMA THERAPIES II AND BIOMARKERS: P.255SUNFISH Part 1: RG7916 treatment results in a sustained increase of SMN protein levels and the first clinical efficacy results in patients with type 2 or 3 SMA
| Autor: | Mercuri, E., Baranello, G., Kirschner, J., Servais, L., Goemans, N., Carmela Pera, M., Buchbjerg, J., Armstrong, G., Kletzl, H., Gerber, M., Czech, C., Cleary, Y., Gorni, K., Khwaja, O. |
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| Zdroj: | In Neuromuscular Disorders October 2018 28 Supplement 2:S108-S108 |
| Databáze: | ScienceDirect |
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