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.
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K. Bushby | F. Muntoni | K. Anthony | G. Dickson | V. Arechavala-Gomeza | R. Kole | M. Wood | S. Wilton | S. Çırak | C. Sewry | V. Straub | M. Guglieri | S. Abbs | S. Torelli | J. Morgan | Lucy Feng | S. Shrewsbury | J. Bourke | Dominic J. Wells | M. Garralda