This open-label study evaluates casimersen and golodirsen in DMD patients with rare single exon duplications (exon 45 or 53). Over 48 weeks, dystrophin levels increased from 0.94% to 5.1%, with dystrophin-positive fibers rising from 14% to 50%. RT-PCR confirmed exon skipping, while pulmonary and cardiac functions remained stable. No major safety concerns emerged, but longer trials with larger cohorts are needed to assess long-term clinical benefits. These findings highlight exon skipping as a targeted therapy for rare DMD mutations.

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