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Genetic strategies for therapy of Duchenne muscular dystrophy.

Mol Ther Nucleic Acids · 2025 · PMC12664987 · PMID 41328300

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135 demonstrated that the primary endpoint change from baseline in the North Star Ambulatory Assessment (NSAA) score did not reach statistical significance between DMD patients after a single intravenous administration of delandistrogene moxeparvovec (1.33 × 10 14 vector genomes per kilogram of body weight; vg/kg; n = 63) and the placebo group ( n = 62).

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