A phase 2/3 randomized clinical trial followed by an open-label extension to evaluate the effectiveness of elamipretide in Barth syndrome, a genetic disorder of mitochondrial cardiolipin metabolism.

Genet Med · 2021 · PMC7935714 · PMID 33077895

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closest p · 0.2× alpha

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a significant trendp < 0.01actually significant

Utilizing a slope model of individual regression lines for each subject to determine the consistent change in indexed stroke volume over the course of exposure, there was a significant trend of an increase in stroke volume over time ( p < 0.01) Supplementary Fig. 4 .

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