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This regulation occurs at multiple levels: Genetic Regulation : Genes encoding the enzymes of the carnitine biosynthetic pathway are subject to transcriptional regulation by factors responsive to metabolic needs
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Additional objectives were to further assess the safety of VPA in children with SMA, to assess performance of selected outcome measures for use in a multi-center clinical trials setting and to look for evidence that would support a biologic effect of VPA in these subjects, such as changes in SMN expression, electrophysiologic outcomes, or both
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