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Some patients experienced developmental delays and other long-term sequelae (e.g

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There were no serious adverse events and no clinically significant adverse changes in platelet count and liver function tests

Promising preliminary results were released in December 2015 for the seven patients who had completed the study up to that point, with an additional patient withdrawing consent

Moreover, a previous study showed that overexpression of CPT1A can reverse the inflammation and fibrosis status of the kidneys by restoring mitochondrial homeostasis [19]