Barely Significant
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Pharmacotherapeutic Approaches to Treatment of Muscular Dystrophies.

Biomolecules · 2023 · PMC10605463 · PMID 37892218

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a nonsignificant trendno p-value reported
A phase 3 placebo-controlled clinical trial of this drug in DMD boys (aged 4–8 years) found it to be safe and tolerated, but functional data from the North Star Ambulatory Assessment (NSAA) criteria showed a nonsignificant trend of improvement in younger patients (<6 years) ( NCT03703882 ) [ 1 ].

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