Blood levels of branched-chain amino acids and alpha-ketoacids in uremic patients given keto analogues of essential amino acids
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In rodents, the enzyme long-chain acyl-CoA dehydrogenase (LCAD) can take over part of the VLCAD function, but in humans LCAD makes little contribution to lcFAO [6]
Finally, key disease phenotypes of the Mmut p.L690Ins/p.L690Ins mice, such as weight loss, were not rescued by BCATi, in contrast with complete rescue in response to replacement of the defective Mmut gene with the normal human MMUT gene via an AAV construct, as shown here and previously in other murine MMA models (37)
Safety profile : Are there potential adverse effects or interactions
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