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With this early rescue dose, the patient likely survives because the dashed black curve in Panel B stays well above the 30% level for functional hepatocytes, which is thought to indicate liver failure

Its strong antioxidant activity is linked to its potential for reducing lipid peroxidation, a process known to contribute to cellular damage and inflammation (87)

The increase of CoA induced by both compounds was smaller than that observed in a genetically corrected patient cell line overexpressing the wild-type PPCS gene (95595-T-PPCS) (Fig

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Their speech development is generally normal or near-normal, and they do not show the early language delays common in HFA