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
Author:
Maria Kinali,Virginia Arechavala-Gomeza,Lucy Feng,Sebahattin Cirak,David Hunt,Carl Adkin,Michela Guglieri,Emma Ashton,Stephen Abbs,Petros Nihoyannopoulos,Maria Elena Garralda,Mary Rutherford,Caroline Mcculley,Linda Popplewell,Ian R Graham et al.
Publication:
The Lancet Neurology
Copyright © 2009 Elsevier Ltd.