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 MD; Virginia Arechavala-Gomeza PhD; Michela Guglieri MD; Lucy Feng PhD; Silvia Torelli PhD; Karen Anthony PhD; Stephen Abbs PhD; Prof Maria Elena Garralda MD; John Bourke MD; Prof Dominic J Wells VetMB PhD; Prof George Dickson PhD; Matthew J A Wood M D PhD; Prof Steve D Wilton PhD; Prof Volker Straub MD; Prof Ryszard Kole PhD; Stephen B Shrewsbury MD; Prof Caroline Sewry PhD; Jennifer E Morgan PhD; Prof Kate Bushby MD; Prof Francesco Muntoni MD

Journal Article

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

MD S
PhD V
MD M
et al.

The Lancet (2011) 378(9791) 595-605

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MD, S. C., PhD, V. A.-G., MD, M. G., PhD, L. F., PhD, S. T., PhD, K. A., … MD, P. F. M. (2011). 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. The Lancet, 378(9791), 595–605.

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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