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Nusinersen in later-onset spinal muscular atrophy: long-term results from the phase 1/2 studies

In: Adam MP, Feldman J, Mirzaa GM, et al., editorsSeattle (WA)University of Washington, Seattle:1993-2024.Available from: [Google Scholar] [5]

Feillet, F., Ogier, H., Cheillan, D., Aquaviva, C., Labarthe, F., Baruteau, J., Chabrol, B., de, Lonlay P., Valayanopoulos, V., Garnotel, R., Dobbelaere, D., Briand, G., Jeannesson, E., Vassault, A., and Vianey-Saban, C