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Johns Hopkins Medicine Research on Drug for Rare Mitochondrial Disorder #mitochondrialdisease
First-of-its-kind therapy developed by Johns Hopkins researchers in the Department of Genetic Medicine cleared for treatment of Barth syndrome, a condition that causes muscle weakness, an enlarged heart, and shortened life expectancy #mitochondrialdisease #johnshopkins https://hub.jhu.edu/2025/09/23/fda-approves-barth-syndrome-treatment/preview/
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