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USA 111 , E426E434 (2014)

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It has been shown that VPA therapeutic metabolites are able to induce the Akt/mTOR/p70S6K pathway through the induction of phosphatidylinositol-3-OH kinase, which leads to a decrease in collagen content and fibrosis in skeletal muscles, a decrease in hind limb contractures, an increase in the integrity of the myocyte sarcolemma, a decrease in the number CD8-positive inflammatory cells, and an increase in the level of activated Akt in the skeletal muscles of experimental animals [13]

The Phase 2b trial (PMID: 23714218) failed to demonstrate significant weight loss compared to placebo over 12 weeks, despite adequate sample size and dosing