Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study
Resource Type
Article
Authors
Kinali, Maria ; Arechavala-Gomeza, Virginia ; Feng, Lucy ; Cirak, Sebahattin ; Hunt, David ; Adkin, Carl ; Guglieri, Michela ; Ashton, Emma ; Abbs, Stephen ; Nihoyannopoulos, Petros ; Garralda, Maria Elena ; Rutherford, Mary ; Mcculley, Caroline ; Popplewell, Linda ; Graham, Ian R ; Dickson, George ; Wood, Matthew JA ; Wells, Dominic J ; Wilton, Steve D ; Kole, Ryszard ; Straub, Volker ; Bushby, Kate ; Sewry, Caroline ; Morgan, Jennifer E ; Muntoni, Francesco
Source
In Lancet Neurology 2009 8(10):918-928
Subject
Primary Research Articles
Language
ISSN
1474-4422