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 TrialLaurent Servais, Frederick J. Schnell, Volker Straub et al.|Nucleic Acid Therapeutics|2021Cited by 128