Long-Term Safety and Efficacy Data of Golodirsen in Ambulatory Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A First-in-human, Multicenter, Two-Part, Open-Label, Phase 1/2 Trial
Laurent Servais(University of Oxford), Frederick J. Schnell(Nationwide Children's Hospital), Valentina Sardone, Claudia Brogna(Università Campus Bio-Medico), Navid Khan(Sarepta Therapeutics (United States)), Volker Straub(Newcastle upon Tyne Hospitals NHS Foundation Trust), A. Dugar(Sarepta Therapeutics (United States)), E. Koenig(Sarepta Therapeutics (United States)), Mariacristina Scoto(University College London), Diane E. Frank(Nationwide Children's Hospital), Cody Akana, Laura Merli, Eugenio Mercuri(Università Cattolica del Sacro Cuore), Cody A. Desjardins(Royal Holloway University of London), Jennifer E. Morgan(Great Ormond Street Hospital), Daniela Leone(Centro Clinico Nemo), Jay S. Charleston(Nationwide Children's Hospital), Marika Pane(Università Cattolica del Sacro Cuore), Giulia Norcia, Chiara Brusa(University of Turin), Gloria Ferrantini, Francesco Muntoni(Great Ormond Street Hospital), Laura Antonaci, Xiaodong Wang(Hebei Agricultural University), Dan Wang(Tianjin University of Traditional Chinese Medicine), Baoguang Han(Sarepta Therapeutics (United States)), Saleh El-Husayni, Andreea Seferian(Sorbonne Université), Mauro Monforte, George Dickson(Royal Holloway University of London), Michela Guglieri(Muscular Dystrophy UK)
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