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
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I. Graham | K. Bushby | L. Popplewell | V. Arechavala-Gomeza | M. Kinali | S. Wilton | S. Çırak | C. Sewry | M. Guglieri | D. Hunt | C. Adkin | Lucy Feng | M. Garralda | Francesco Muntoni | Emma Ashton | Stephen Abbs | Petros Nihoyannopoulos | Mary Rutherford | Caroline Mcculley | George Dickson | Matthew JA Wood | Ryszard Kole | Volker Straub | Jennifer E Morgan | Dominic J. Wells