Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

S. Cirak, V. Arechavala-Gomeza, M. Guglieri, L. Feng, S. Torelli, K. Anthony, S. Abbs, M.E. Garralda, J. Bourke, D.J. Wells, G. Dickson, M.J.A. Wood, Steve Wilton, V. Straub, R. Kole, S.B. Shrewsbury, C. Sewry, J.E. Morgan, K. Bushby, F. Muntoni

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