Metformin as a potential treatment for a rare pediatric disorder

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Northerner

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A study in human skin cells and mice shows the widely-used diabetes drug metformin reduces toxic acid levels in Maple Syrup Urine Disease. The discovery offers the possibility of a new treatment for a disorder identified 1 in 180,000 births. Maple Syrup Urine Disease is a rare inherited disorder involving the dysfunction of an enzyme which breaks down three essential amino acids. Left untreated, infants die shortly after birth. Those diagnosed early can survive, but are forced to eat a very controlled diet. The only treatment is a liver transplant. Researchers show that metformin reduces the toxic acid levels associated with MSUD in both skin cells derived from MSUD patients and in mice.

https://www.sciencedaily.com/releases/2016/07/160706114414.htm

Hehe! Wonderdrug strikes again! 😱 🙂
 
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