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

Autores: Balwani, M.; Gouya, L.; Rees, D.; Stein, P.; Stolzel, U.; Aguilera, P.; Bissell, D. M.; Bonkovsky, H.; Keel, S.; Parker, C.; Philips, J.; Silver, S.; Windyga, J.; D'Avola, Delia; Ross, G.; Stewart, P.; Ritchie, B.; Oh, J.; Harper, P.; Wang, J. D.; Langendonk, J.; Ivanova, A. ; Horie, Y.; Anderson, K. ; Ventura, P.; Chan, A.; Penz, C.; Simon, A. ; Dinh, Q. ; Liu, G. ; Sardh, E.