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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author: Francesco Muntoni, Sebahattin Çırak, Maria Elena Garralda, Virginia Arechavala-Gomeza, Karen Anthony, Dominic J Wells, Volker Straub, Ryszard Kole, Michela Guglieri, Lucy Feng, Silvia Torelli, Caroline A Sewry, Matthew J. Wood, John Bourke
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