Targeting porphyria
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DOI: 10.1038/s41591-020-0987-0
Abstract: A phase 3 trial of givosiran, an RNA-interference therapy, for acute intermittent porphyria resulted in a reduced rate of porphyria attacks. Acute hepatic porphyria is a set of rare diseases in which porphyrins build up,… read more here.
Keywords: medicine; targeting porphyria; porphyria;