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A Clinical Trial of a Methylmalonic Acidemia (MMA) Due to MUT Deficiency Treatment for Children and Adults

A Clinical Trial of a Propionic Acidemia (PA) Treatment for Children and Adults


Inherited metabolic disorders occur when a genetic mutation leads to a missing or dysfunctional version of a protein that is important for metabolism. While certain lifestyle changes, like dietary restrictions, may help alleviate the most severe symptoms, there are no treatments for the underlying genetic cause for many of these disorders.
Moderna is working to address this underlying cause by providing new instructions to cells so the body can replace these missing or dysfunctional proteins. These instructions are known as messenger RNA or mRNA. Our cells use mRNA to interpret information passed down through our genes and turns this information into proteins that build or regulate our bodies. 

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