20-11-2019 17:15 via medpagetoday.com

FDA OKs First Agent for Rare Genetic Disease

]]>(MedPage Today) -- WASHINGTON--The FDA approved the RNA interference (RNAi) drug givosiran (Givlaari) on Wednesday, the first-ever treatment for treating adults with acute hepatic porphyria, a painful inherited disorder where toxic porphyrin...
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