Gene-silencing technology gets first drug approval after 20-year wait - Nature.com

Nature.com
Gene-silencing technology gets first drug approval after 20-year wait
Nature.com
US regulators have approved the first therapy based on RNA interference (RNAi), a technique that can be used to silence specific genes linked to disease. The drug, patisiran, targets a rare condition that can impair heart and nerve function. The ...
Designating an Orphan Product: Drugs and Biological Products - FDAFDA
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