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Moderna reports ‘encouraging’ early data for one of its rare disease medicines

Moderna said Friday that one of its rare disease drug programs showed promise in its first clinical trials, according to an early look at the data.

The drug is meant to treat propionic acidemia, an uncommon genetic disorder that causes the body to not have enough of an enzyme essential for breaking down proteins. Estimates about how many people have this disorder vary, but those who do have difficulty processing certain foods and, early into life, will display symptoms like trouble eating, vomiting, dehydration and lethargy.

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