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Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-esclation study
Journal Article
Cirak, S., Arechavala-Gomeza, V., Guglieri, M., Feng, L., Torelli, S., Anthony, K., Abbs, S., Garralda, M. E., Wells, D., Dickson, G., Wood, M. J. A., Wilton, S. D., Straub, V., Shrewsbury, S., Sewry, C., Morgan, J. E., Bushby, K., & Muntoni, F. Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-esclation study. https://doi.org/10.1016/S0140-6736%2811%2960756-3