Metformin as a potential treatment for a rare pediatric disorder

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.
Go to Source