Overall Health, Daily Functioning, and Quality of Life in Acute Hepatic Porphyria Patients: ENVISION, a Phase 3 Global, Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial

Autores: Sardh, E. ; Gouya, L.; Rees, D. C.; Stein, P. ; Stolzel, U.; Peiro, P. A. ; Bissell, D. M. ; Bonkovsky, H. L.; Keel, S. ; Parker, C.; Phillips, J. D.; Silver, S.; Windyga, J. ; D'Avola, Delia; Ross, G.; Stewart, P.; Ritchie, B.; Oh, J.; Harper, P.; Wang, J. D.; Langendonk, J. G.; Ivanova, A.; Horie, Y.; Anderson, K. E. ; Ventura, P.; Kauppinen, R. ; Vassiliou, D.; Wang, B.; Hother-Nielsen, O.; Nakahara, T.; Lee, M. J. ; Sasapu, A.; Scalera, S. ; Lin, T. ; Penz, C.; Simon, A.; Ko, J. ; Balwani, M.