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PPMO-based exon skipping therapy improves respiratory function in the <i>mdx</i> mouse model of Duchenne muscular dystrophy.

Mol Ther Nucleic Acids · 2026 · PMC12809079 · PMID 41552386

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showed a trendp < 0.05actually significant
100 mg/kg PPMO-treated mice), while lower (30 mg/kg) and intermittent doses (50 mg/kg) showed a trend toward improved strength ( p < 0.05, WT and untreated mdx vs. lower and intermittent dose PPMO-treated mdx mice) ( Figure 6 A).

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