Encore - a Randomized, Controlled, Open-Label Non-Inferiority Study Comparing Eliglustat to Imiglucerase in Gaucher Disease Type 1 Patients Stabilized on Enzyme Replacement Therapy... von Rosenbloom, Barry E., Cox, Timothy M., Drelichman, Guillermo I., Cravo, Renata, Balwani, Manisha, Burrow, Thomas Andrew, Martins, Ana Maria, Lukina, Elena, Ross, Leorah H., Angell, Jennifer, Peterschmitt, Judith

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Structure and Expression of Novel Spliced Leader RNA Genes in Caenorhabditis elegans(∗) von Ross, Leorah H., Freedman, Jonathan H., Rubin, Charles S.

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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: Res... von Amato, Dominick J, Dasouki, Majed, Packman, Seymour, Pastores, Gregory M., Assouline, Sarit, Balwani, Manisha, Mistry, Pramod, Ortega, Andres, Shankar, Suma, Solano, Maria Helena, Ross, Leorah H., Angell, Jennifer, Peterschmitt, Judith

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Encore: A Randomized, Controlled, Open-Label Non-Inferiority Study Comparing Eliglustat To Imiglucerase In Gaucher Disease Type 1 Patients On Enzyme Replacement Therapy Who Have Re... von Andrew Burrow, Thomas, Balwani, Manisha, Cox, Timothy M., Drelichman, Guillermo, Cravo, Renata, Martins, Ana Maria, Lukina, Elena, Rosenbloom, Barry E., Ross, Leorah H., Angell, Jennifer, Puga, Ana Cristina

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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: 9 Mo... von Shankar, Suma, Lukina, Elena, Amato, Dominick J, Dasouki, Majed, Packman, Seymour, Pastores, Gregory M, Assouline, Sarit, Balwani, Manisha, Mistry, Pramod K, Ross, Leorah H, Marulkar, Sachin, Peterschmitt, M. Judith

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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: Res... von Mistry, Pramod, Amato, Dominick J., Dasouki, Majed, Packman, Seymour, Pastores, Gregory M., Assouline, Sarit, Balwani, Manisha, Ortega, Andres, Shankar, Suma, Solano, Maria Helena, Ross, Leorah H., Angell, Jennifer, Peterschmitt, M. Judith

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ENCORE, a randomized, controlled, open-label non-inferiority study comparing eliglustat to imiglucerase in Gaucher disease type 1 patients stabilized on enzyme replacement therapy:... von Cox, Timothy M., Drelichman, Guillermo I., Cravo, Renata, Balwani, Manisha, Burrow, Thomas Andrew, Martins, Ana Maria, Lukina, Elena, Rosenbloom, Barry E., Ross, Leorah H., Angell, Jennifer, Peterschmitt, M. Judith

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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: Res... von Mistry, Pramod, Amato, Dominick J., Dasouki, Majed, Packman, Seymour, Pastores, Gregory M., Assouline, Sarit, Balwani, Manisha, Ortega, Andres, Shankar, Suma, Solano, Maria Helena, Ross, Leorah H., Angell, Jennifer, Peterschmitt, M. Judith

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Eliglustat, an Investigational Oral Therapy for Gaucher Disease Type 1: Phase 2 Results After 4 Years of Treatment von Peterschmitt, M. Judith, Lukina, Elena, Watman, Nora, Dragosky, Marta, Pastores, Gregory M, Arreguin, Elsa Avila, Rosenbaum, Hanna, Zimran, Ari, Sysoeva, Elena, Aguzzi, Rasha, Ross, Leorah H, Puga, Ana Cristina

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Improvements in Skeletal Manifestations in Gaucher Disease Type 1 Patients After 3 Years of Treatment with Oral Eliglustat During a Phase 2 Trial von Peterschmitt, M. Judith, Kamath, Ravi S, Lukina, Elena, Watman, Nora, Dragosky, Marta, Iastrebner, Marcelo, Pastores, Gregory M, Arreguin, Elsa Avila, Phillips, Mici, Rosenbaum, Hanna, Sysoeva, Elena, Aguzzi, Rasha, Ross, Leorah H, Puga, Ana Cristina, Rosenthal, Daniel I

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Eliglustat, An Investigational Oral Therapy for Gaucher Disease Type 1: Phase 2 Results After 3 Years von Peterschmitt, M. Judith, Lukina, Elena, Watman, Nora, Dragosky, Marta, Iastrebner, Marcelo, Pastores, Gregory, Arreguin, Elsa Avila, Phillips, Mici, Rosenbaum, Hanna, Sysoeva, Elena, Aguzzi, Rasha, Ross, Leorah H, Puga, Ana Cristina

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Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia von Locatelli, Franco, Lang, Peter, Wall, Donna, Meisel, Roland, Corbacioglu, Selim, Li, Amanda M., de la Fuente, Josu, Shah, Ami J., Carpenter, Ben, Kwiatkowski, Janet L., Mapara, Markus, Liem, Robert I., Cappellini, Maria Domenica, Algeri, Mattia, Kattamis, Antonis, Sheth, Sujit, Grupp, Stephan, Handgretinger, Rupert, Kohli, Puja, Shi, Daoyuan, Ross, Leorah, Bobruff, Yael, Simard, Christopher, Zhang, Lanju, Morrow, Phuong Khanh, Hobbs, William E., Frangoul, Haydar

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Angiopoietin-1 protects the adult vasculature against plasma leakage von Thurston, Gavin, Rudge, John S., Ioffe, Ella, Zhou, Hao, Ross, Leorah, Croll, Susan D., Glazer, Nicole, Holash, Jocelyn, McDonald, Donald M., Yancopoulos, George D.

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