NIH researchers identify therapy that may curb kidney deterioration in patients with rare disorder

Innovation in mouse model helps researchers distinguish disease mechanisms and biomarkers

A team led by researchers at the National Institutes of Health has overcome a major biological hurdle in an effort to find improved treatments for patients with a rare disease called methylmalonic acidemia (MMA). Using genetically engineered mice created for their studies, the team identified a set of biomarkers of kidney damage — a hallmark of the disorder — and demonstrated that antioxidant therapy protected kidney function in the mice.

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This page was last updated on Friday, January 21, 2022