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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Journal

The Lancet

Topics

Chemistry

Biochemistry

Medicine

Internal Medicine

Organic Chemistry

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DOI

10.1016/s0140-6736(11)60756-3

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