DOI: 10.1055/s-00000071

Seminars in Neurology

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Cirak S, Arechavala-Gomeza V, Guglieri M , et al.
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.

Lancet 2011;
378 (9791) 595-605

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