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4. Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria. Balwani M; Sardh E; Ventura P; Peiró PA; Rees DC; Stölzel U; Bissell DM; Bonkovsky HL; Windyga J; Anderson KE; Parker C; Silver SM; Keel SB; Wang JD; Stein PE; Harper P; Vassiliou D; Wang B; Phillips J; Ivanova A; Langendonk JG; Kauppinen R; Minder E; Horie Y; Penz C; Chen J; Liu S; Ko JJ; Sweetser MT; Garg P; Vaishnaw A; Kim JB; Simon AR; Gouya L; N Engl J Med; 2020 Jun; 382(24):2289-2301. PubMed ID: 32521132 [TBL] [Abstract][Full Text] [Related]
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