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

Sebahattin Cirak, Virginia Arechavala-Gomeza, Michela Guglieri, Lucy Feng, Silvia Torelli, Karen Anthony, Stephen Abbs, Maria Elena Garralda, John Bourke, Dominic J. Wells, George Dickson, Matthew Ja Wood, Steve D. Wilton, Volker Straub, Ryszard Kole, Stephen B. Shrewsbury, Caroline Sewry, Jennifer E. Morgan, Kate Bushby, Francesco Muntoni

Research output: Contribution to Journal › Article › peer-review

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