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
2019; Elsevier BV; Volume: 70; Issue: 1 Linguagem: Inglês
10.1016/s0618-8278(19)30142-2
ISSN1600-0641
AutoresManisha 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,
Tópico(s)Folate and B Vitamins Research
Referência(s)