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.

Although this change did not reach statistical significance, a previous trial on an unsuccessful DMD treatment showed a 1.2% increase [confidence interval (95% CI) −1.1 to 3.4] in myofibers positive for fetal/developmental myosin over 48 weeks [ 33 ].

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