Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study
Sebahattin Çirak(University College London), Virginia Arechavala‐Gomeza(University College London), Michela Guglieri(Newcastle University), Lucy Feng(University College London), Silvia Torelli(University College London), Karen Anthony(University College London), Stephen Abbs(Guy's Hospital), Maria Elena Garralda, John Bourke(Newcastle University), Dominic J. Wells(Royal Veterinary College), George Dickson(Royal Holloway University of London), Matthew J. A. Wood(University of Oxford), Steve D. Wilton(University of Western Australia), Volker Straub(Newcastle University), Ryszard Kole(Sarepta Therapeutics (United States)), Stephen B. Shrewsbury(Sarepta Therapeutics (United States)), Caroline A. Sewry(University College London), Jennifer E. Morgan(University College London), Kate Bushby(Newcastle University), Francesco Muntoni(University College London)
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