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Impact of elosulfase alfa on pain in patients with Morquio syndrome type A
Harmatz, Paul; Treadwell, M; Burton, Barbara K; Mitchell, John; Muschol, N; Jones, Simon; Pastores, Gregory; Lau, Heather; Sparkes, R; Sutton, VR; Genter, F; Haller, C; Shaywitz, A
2015-03-13; 1096-7206,Molecular genetics & metabolism - id: 1486682, year: 2015

Effect of oral eliglustat on splenomegaly in patients with Gaucher disease type 1: the ENGAGE randomized clinical trial
Mistry, Pramod K; Lukina, Elena; Ben Turkia, Hadhami; Amato, Dominick; Baris, Hagit; Dasouki, Majed; Ghosn, Marwan; Mehta, Atul; Packman, Seymour; Pastores, Gregory; Petakov, Milan; Assouline, Sarit; Balwani, Manisha; Danda, Sumita; Hadjiev, Evgueniy; Ortega, Andres; Shankar, Suma; Solano, Maria Helena; Ross, Leorah; Angell, Jennifer; Peterschmitt, M Judith
2015-02-23; 0098-7484,JAMA - id: 1466742, year: 2015 Journal Article; Research Support, Non-U.S. Gov't

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 18 months
Mistry, Pramod; Amato, Dominick J; Dasouki, Majed; Packman, Seymour; Pastores, Gregory M; Assouline, Sarit; Balwani, Manisha; Ortega, Andres; Shankar, Suma; Solano, Maria Helena; Rossk, Leorah H; Angell, Jennifer; Peterschmitt, MJudith
2015-03-13; 1096-7206,Molecular genetics & metabolism - id: 1486702, year: 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 18 Months
Amato, Dominick J; Dasouki, Majed; Packman, Seymour; Pastores, Gregory M; Assouline, Sarit; Balwani, Manisha; Mistry, Pramod; Ortega, Andres; Shankar, Suma; Helena Solano, Maria; Ross, Leorah H; Angell, Jennifer; Peterschmitt, Judith
2015-03-19; 1528-0020,Blood - id: 1497732, year: 2014

Safety and efficacy of long-term enzyme replacement therapy with velaglucerase alfa in patients with type 1 Gaucher disease transitioned from imiglucerase
Elstein, Deborah; Giraldo, Pilar; Mehta, Atul; Pastores, Gregory; Rhead, William; Smith, Laurie; Wang, Nan; Crombez, Eric; Zimran, Ari
2014-03-14; 1096-7192,Molecular genetics & metabolism - id: 833382, year: 2014