Individual patient responses to eliglustat in treatment-naïve adults with Gaucher disease type 1: Final data from the phase 3 ENGAGE trial
Mistry P, Lukina E, Turkia H, Shankar S, Feldman H, Ghosn M, Mehta A, Packman S, Lau H, Petakov M, Assouline S, Balwani M, Danda S, Hadjiev E, Ortega A, Foster M, Gaemers S, Peterschmitt M. Individual patient responses to eliglustat in treatment-naïve adults with Gaucher disease type 1: Final data from the phase 3 ENGAGE trial. Molecular Genetics And Metabolism 2020, 129: s110-s111. DOI: 10.1016/j.ymgme.2019.11.284.Peer-Reviewed Original Research