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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

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

Multi-domain impact of elosufase alfa in Morquio A syndrome in the pivotal phase III trial
Hendriksz, Christian J; Giugliani, Roberto; Harmatz, Paul; Mengel, Eugen; Guffon, Nathalie; Valayannopoulos, Vassili; Parini, Rossella; Hughes, Derralynn; Pastores, Gregory M; Lau, Heather A; Al-Sayed, Moeenaldeen D; Raiman, Julian; Yang, Ke; Mealiffe, Matthew; Haller, Christine
2014-10-13; 1096-7192,Molecular genetics & metabolism - id: 1299642, year: 2014 JOURNAL ARTICLE

Skeletal improvement in patients with Gaucher disease type 1: a phase 2 trial of oral eliglustat
Kamath, Ravi S; Lukina, Elena; Watman, Nora; Dragosky, Marta; Pastores, Gregory M; Arreguin, Elsa Avila; Rosenbaum, Hanna; Zimran, Ari; Aguzzi, Rasha; Puga, Ana Cristina; Norfleet, Andrea M; Peterschmitt, M Judith; Rosenthal, Daniel I
2014-05-13; 0364-2348,Skeletal radiology - id: 978472, year: 2014 JOURNAL ARTICLE

Eliglustat, an investigational oral therapy for Gaucher disease type 1: Phase 2 trial results after 4years of treatment
Lukina, Elena; Watman, Nora; Dragosky, Marta; Pastores, Gregory M; Arreguin, Elsa Avila; Rosenbaum, Hanna; Zimran, Ari; Angell, Jennifer; Ross, Leorah; Puga, Ana Cristina; Peterschmitt, Judith M
2014-05-22; 1079-9796,Blood cells, molecules, & diseases - id: 1003472, year: 2014 JOURNAL ARTICLE