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Balwani, Manisha; Sardh, Eliane; Ventura, Paolo; Peiró, Paula Aguilera; Rees, David C; Stölzel, Ulrich; Bissell, D. Montgomery; Bonkovsky, Herbert L; Windyga, Jerzy; Anderson, Karl E; Parker, Charles; Silver, Samuel M; Keel, Siobán B; Wang, Jiaan-Der; Stein, Penelope E; Harper, Pauline; Vassiliou, Daphne; Wang, Bruce; Phillips, John; Ivanova, Aneta; Langendonk, Janneke G; Kauppinen, Raili; Minder, Elisabeth; Horie, Yutaka; Penz, Craig; Chen, Jihong; Liu, Shangbin; Ko, John J; Sweetser, Marianne T; Garg, Pushkal; Vaishnaw, Akshay; Kim, Jae B; Simon, Amy R; Gouya, Laurent
The New England journal of medicine, 06/2020, Letnik: 382, Številka: 24Journal Article
In a randomized phase 3 trial involving patients with acute intermittent porphyria, the use of givosiran, an oligonucleotide drug designed to target messenger RNA encoding aminolevulinic acid synthase, led to a 74% lower annualized porphyria attack rate than the use of placebo at 6 months.
