Skip to main content

Research Repository

Advanced Search

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; Arechavala-Gomeza, V; Guglieri, M; Feng, L; Torelli, S; Anthony, K; Abbs, S; Garralda, M E; Wells, DJ; Dickson, G; Wood, M J A; Wilton, S D; Straub, V; Shrewsbury, S; Sewry, C; Morgan, J E; Bushby, K; Muntoni, F

Authors

S Cirak

V Arechavala-Gomeza

M Guglieri

L Feng

S Torelli

K Anthony

S Abbs

M E Garralda

DJ Wells

G Dickson

M J A Wood

S D Wilton

V Straub

S Shrewsbury

C Sewry

J E Morgan

K Bushby

F Muntoni



Citation

Cirak, S., Arechavala-Gomeza, V., Guglieri, M., Feng, L., Torelli, S., Anthony, K., …Muntoni, F. 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. https://doi.org/10.1016/S0140-6736%2811%2960756-3

Journal Article Type Article
Deposit Date Nov 12, 2014
Journal Lancet
Volume 378
Issue 9791
Pages 595-605
DOI https://doi.org/10.1016/S0140-6736%2811%2960756-3
Public URL https://rvc-repository.worktribe.com/output/1417748
Additional Information Corporate Creators : Guy's Hospital, London, Imperial College London, Newcastle, Oxford, RJAH Orthopaedic Hospital, Royal Holloway, UCL, Western Australia




You might also like



Downloadable Citations