2017
Long-term results of ENGAGE: a phase 3, randomized, double‑blind, placebo-controlled, multi‑center study investigating the efficacy and safety of eliglustat in adults with type 1 Gaucher disease
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, Wu Y, Gaemers S, Peterschmitt M. Long-term results of ENGAGE: a phase 3, randomized, double‑blind, placebo-controlled, multi‑center study investigating the efficacy and safety of eliglustat in adults with type 1 Gaucher disease. Molecular Genetics And Metabolism 2017, 120: s97-s98. DOI: 10.1016/j.ymgme.2016.11.243.Peer-Reviewed Original Research
2015
ENGAGE — A phase 3, randomized, double-blind, placebo-controlled, multi-center study to investigate the efficacy and safety of eliglustat in adults with Gaucher disease type 1: Results after 18months
Mistry P, Amato D, Dasouki M, Packman S, Pastores G, Assouline S, Balwani M, Ortega A, Shankar S, Solano M, Ross L, Angell J, Peterschmitt M. ENGAGE — A phase 3, randomized, double-blind, placebo-controlled, multi-center study to investigate the efficacy and safety of eliglustat in adults with Gaucher disease type 1: Results after 18months. Molecular Genetics And Metabolism 2015, 114: s81-s82. DOI: 10.1016/j.ymgme.2014.12.179.Peer-Reviewed Original Research
2013
A phase 3, randomized, double-blind, placebo-controlled, multi-center study to investigate the efficacy and safety of eliglustat in patients with Gaucher disease type 1 (ENGAGE): Results after 9months of treatment
Mistry P, Lukina E, Dridi M, Amato D, Baris H, Dasouki M, Ghosn M, Mehta A, Packman S, Pastores G, Petakov M, Peterschmitt M. A phase 3, randomized, double-blind, placebo-controlled, multi-center study to investigate the efficacy and safety of eliglustat in patients with Gaucher disease type 1 (ENGAGE): Results after 9months of treatment. Molecular Genetics And Metabolism 2013, 108: s66-s67. DOI: 10.1016/j.ymgme.2012.11.171.Peer-Reviewed Original Research