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Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-esclation, proof-of-concept study

Kinali, M; Arechavala-Gomeza, V; Feng, L; Cirak, S; Hunt, D; Adkin, C; Guglieri, M; Ashton, E; Abbs, S; Nihoyannopoulos, P; Garralda, M E; Rutherford, M; McCulley, C; Popplewell, L; Graham, I R; Dickson, G; Wood, M J; Wells, D J; Wilton, S D; Kole, R; Staub, V; Bushby, K; Sewry, C; Morgan, J E; Muntoni, F

Authors

M Kinali

V Arechavala-Gomeza

L Feng

S Cirak

D Hunt

C Adkin

M Guglieri

E Ashton

S Abbs

P Nihoyannopoulos

M E Garralda

M Rutherford

C McCulley

L Popplewell

I R Graham

G Dickson

M J Wood

D J Wells

S D Wilton

R Kole

V Staub

K Bushby

C Sewry

J E Morgan

F Muntoni



Citation

Kinali, M., Arechavala-Gomeza, V., Feng, L., Cirak, S., Hunt, D., Adkin, C., …Muntoni, F. (2009). Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-esclation, proof-of-concept study. https://doi.org/10.1016/S1474-4422%2809%2970211-X

Journal Article Type Article
Acceptance Date Aug 19, 2009
Publication Date Oct 1, 2009
Deposit Date Nov 12, 2014
Publicly Available Date Nov 30, 2018
Journal Lancet Neurology
Peer Reviewed Peer Reviewed
Volume 8
Issue 10
Pages 918-28
DOI https://doi.org/10.1016/S1474-4422%2809%2970211-X
Public URL https://rvc-repository.worktribe.com/output/1424491
Additional Information Corporate Creators : Guy's Hospital, London, Hammersmith Hospital, Imperial College London, Institute of Child Health, London, Newcastle, St Mary's Hospital, London

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