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New mRNA therapy shows promise in treating ‘ultrarare’ inherited disease


A new treatment may be able to treat a life-threatening disorder that predominantly affects children, initial findings from a pioneering clinical trial suggest.

The disorder, called propionic acidemia (PA), is an inherited metabolic disorder that affects around 1 in 100,000 people in the U.S. Individuals with the disease have faulty copies of genes needed to make parts of the enzyme propionyl-coenzyme A carboxylase (PCC), which breaks down the building blocks of certain proteins and fats. 



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