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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

Abstract:: We report clinical safety and biochemical efficacy from a dose-ranging study of intravenously administered AVI-4658 phosphorodiamidate morpholino oligomer (PMO) in patients with Duchenne muscular dystrophy. We undertook an open-label, phase 2, dose-escalation study (0·5, 1·0, 2·0, 4·0, 10·0, and 20·0 mg/kg bodyweight) in ambulant patients with Duchenne muscular dystrophy aged 5-15 years with amenable deletions in DMD. Participants had a muscle biopsy before starting treatment and after 12 wee...
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APA Style

Cirak, S., Arechavala-Gomeza, V., Guglieri, M., Feng, L., Torelli, S., Anthony, K., Abbs, S., Garralda, M., Bourke, J., Wells, D., Dickson, G., Wood, M., Wilton, S., Straub, V., Kole, R., Shrewsbury, S., Sewry, C., Morgan, J., Bushby, K., & 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-escalation study. Lancet, 378(9791), 595–605.

MLA Style

Cirak, S., 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, vol. 378, no. 9791, 2011, pp. 595–605.

Chicago Style

Cirak, S, V Arechavala-Gomeza, M Guglieri, L Feng, S Torelli, K Anthony, S Abbs, et al. 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.” Lancet 378 (9791): 595–605.
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Publisher copy:: 10.1016/S0140-6736(11)60756-3

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+ Arechavala-Gomeza, V More by this author

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+ Guglieri, M More by this author

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+ Feng, L More by this author

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+ Torelli, S More by this author

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Bibliographic Details

Journal:: Lancet
Volume:: 378
Issue:: 9791
Pages:: 595-605
Publication date:: 2011-08-13
DOI:: 10.1016/S0140-6736(11)60756-3
EISSN:: 1474-547X
ISSN:: 0140-6736

Item Description

Language:: English
Pubs id:: pubs:179417
UUID:: uuid:64d3656f-828d-4739-bd87-40206502d329
Local pid:: pubs:179417
Source identifiers:: 179417
Deposit date:: 2012-12-19

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Copyright date:: 2011

Licence:: Terms and Conditions of Use for Oxford University Research Archive

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