TrialNucleic acid therapeutics2022
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 et al.PubMed ↗Full text ↗Publisher ↗
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