A research group led by Dr. Kenneth MacLean at the University of Colorado has shown that a combination of formate treatment with betaine could dramatically improve clinical outcome in Homocystinuria (HCU), a rare metabolic condition characterized by an excess of homocysteine in the urine. This new treatment removes the need for a methionine restricted diet. Treatment of HCU mice with sodium formate alone effectively halved plasma homocysteine levels, and the combination of this treatment with betaine results in the lowest levels of homocysteine ever observed in an animal model of the disease.
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