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
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E. Mercuri | F. Muntoni | L. Servais | M. Scoto | V. Straub | M. Guglieri | D. Leone | Xiaodong Wang | A. Seferian | A. Dugar | E. Koenig | N. Khan | B. Han | Dan Wang