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Trial tests Covid vaccine technology against a rare disease that affects babies

Preliminary study of messenger RNA therapy against propionic acidaemia shows it is safe, but doubts remain about its usefulness ....

Dwight Koeberl , R Jude Samulski , Gloria Gonz , Duke University Hospital , Institute For Rare Diseases Research , University Of Navarre , Jude Samulski , Applied Medical Research , Gene Therapy Unit , Rare Diseases Research ,

New mRNA therapy shows promise in treating 'ultrarare' inherited disease

Initial trial results suggest that a new mRNA therapy may be able to safely and effectively treat propionic acidemia, a rare metabolic disorder. ....

Duke University , North Carolina , United States , Dwight Koeberl , Pawel Swietach , University Of Oxford , Duke University School Of Medicine , Live Science , Duke University School ,