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

Cirak, S and Arechavala-Gomeza, V and Guglieri, M and Feng, L and Torelli, S and Anthony, K and Abbs, S and Garralda, M E and Wells, DJ and Dickson, G and Wood, M J A and Wilton, S D and Straub, V and Shrewsbury, S and Sewry, C and Morgan, J E and Bushby, K and Muntoni, F (2011) Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-esclation study. Lancet, 378 (9791). pp. 595-605.

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