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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Published in Lancet Neurol on August 25, 2009

Authors

Maria Kinali1, 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, George Dickson, Matthew J A Wood, Dominic J Wells, Steve D Wilton, Ryszard Kole, Volker Straub, Kate Bushby, Caroline Sewry, Jennifer E Morgan, Francesco Muntoni

Author Affiliations

1: The Dubowitz Neuromuscular Centre, University College London Institute of Child Health London, London, UK.

Associated clinical trials:

Dose-Ranging Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy (DMD) Patients | NCT00844597

Safety and Efficacy Study of Antisense Oligonucleotides in Duchenne Muscular Dystrophy | NCT00159250

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