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Rare Disease Therapy

All articles tagged with #rare disease therapy

"Moderna's mRNA Breakthrough in Rare-Disease Trial"
health-and-medicine2 years ago

"Moderna's mRNA Breakthrough in Rare-Disease Trial"

Moderna, known for its Covid-19 vaccine, is making progress in developing therapies using mRNA, with early study results showing promise for a rare-disease therapy targeting propionic acidemia. The treatment demonstrated potential in reducing life-threatening metabolic emergencies in some participants, indicating progress in Moderna's efforts to turn mRNA into medicines.