GS-14-ENVISION, a phase 3 study to evaluate efficacy and safety of givosiran, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1, in acute hepatic porphyria patients
医学
试验药物
RNA干扰
药理学
ATP合酶
内科学
化学
基因
临床试验
生物化学
核糖核酸
作者
Manisha Balwani,Laurent Gouya,David Rees,Penelope E. Stein,Ulrich Stölzel,Paula Aguilera,D. Montgomery Bissell,Herbert L. Bonkovsky,Sioḃán Keel,Charles Parker,John Phillips,Samuel Silver,Jerzy Windyga,Delia D’Avola,Gayle Ross,Peter Stewart,Bruce Ritchie,Jeeyoung Oh,Pauline Harper,Jiaan‐Der Wang,Janneke G. Langendonk,Aneta Ivanova,Yutaka Horie,Karl E. Anderson,Paolo Ventura,Amy B. W. Chan,Craig Penz,Amy Simon,Quinn Dinh,Gary Liu,Eliane Sardh
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(2025-6-4)
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