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The compound has been investigated in preclinical research across cardiac, skeletal muscle, neuronal, and renal models, and has progressed through multiple Phase 3 clinical trials in Barth syndrome and primary mitochondrial myopathy
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Am J Med 118(5):2734 Polonsky W, Fisher L, Hessler D, Bruhn D, Best J (2011) Patient perspectives on once-weekly medications for diabetes
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