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Journal Articles

Type II NKT-T FH cells against Gaucher lipids regulate B-cell immunity and inflammation

Shiny Nair, Chandra Sekhar Boddupalli, Rakesh Verma, Jun Liu, Ruhua Yang, Gregory M. Pastores, Pramod K. Mistry, Madhav V. Dhodapkar

Blood (2015) 125 (8): 1256–1271.

https://doi.org/10.1182/blood-2014-09-600270

Published: 2015

Includes: Supplemental data

Journal Articles

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

Dominick J Amato, MD, Majed Dasouki, MD, Seymour Packman, MD PhD, Gregory M. Pastores, MD, Sarit Assouline, MD MSc, Manisha Balwani, MD MS, Pramod Mistry, MD PhD FRCP, Andres Ortega, MD, Suma Shankar, MD PhD, Maria Helena Solano, MD, Leorah H. Ross, MD PhD, Jennifer Angell, ScM, Judith Peterschmitt, MD

Blood (2014) 124 (21): 2732.

https://doi.org/10.1182/blood.V124.21.2732.2732

Published: 2014

Journal Articles

Type II NKT-TFH Cells Against Gaucher Lipids Regulate B Cell Immunity and Inflammation

Shiny Nair, PhD, Chandra Sekhar Boddupalli, PhD, Rakesh Verma, PhD, Jun Liu, Yang Ruhua, Gregory M Pastores, MD, Pramod Mistry, MD PhD FRCP, Madhav V. Dhodapkar, MD

Blood (2014) 124 (21): 755.

https://doi.org/10.1182/blood.V124.21.755.755

Published: 2014

Journal Articles

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 Month Results

Suma Shankar, MD, PhD, Elena Lukina, MD, PhD, Dominick J Amato, MD, Majed Dasouki, MD, Seymour Packman, MD, PhD, Gregory M Pastores, MD, Sarit Assouline, MD, Manisha Balwani, MD, MS, Pramod K Mistry, MD, PhD, FRCP, Leorah H Ross, MD, PhD, Sachin Marulkar, MBBS, MPH, M. Judith Peterschmitt, MD

Blood (2013) 122 (21): 2275.

https://doi.org/10.1182/blood.V122.21.2275.2275

Published: 2013

Journal Articles

Eliglustat, an Investigational Oral Therapy for Gaucher Disease Type 1: Phase 2 Results After 4 Years of Treatment

M. Judith Peterschmitt, MD, Elena Lukina, PhD, MD, Nora Watman, MD, Marta Dragosky, M.D., Gregory M Pastores, Elsa Avila Arreguin, M.D., Hanna Rosenbaum, MD, Ari Zimran, MD, Elena Sysoeva, Rasha Aguzzi, Leorah H Ross, Ana Cristina Puga, M.D.

Blood (2012) 120 (21): 1038.

https://doi.org/10.1182/blood.V120.21.1038.1038

Published: 2012

Journal Articles

Improvements in Skeletal Manifestations in Gaucher Disease Type 1 Patients After 3 Years of Treatment with Oral Eliglustat During a Phase 2 Trial,

M. Judith Peterschmitt, MD, Ravi S Kamath, MD, Elena Lukina, PhD, MD, Nora Watman, MD, Marta Dragosky, M.D., Marcelo Iastrebner, M.D., Gregory M Pastores, Elsa Avila Arreguin, M.D., Mici Phillips, M.D., Hanna Rosenbaum, MD, Elena Sysoeva, Rasha Aguzzi, Leorah H Ross, Ana Cristina Puga, M.D., Daniel I Rosenthal, M.D.

Blood (2011) 118 (21): 3216.

https://doi.org/10.1182/blood.V118.21.3216.3216

Published: 2011

Journal Articles

Report on the Safety of Velaglucerase Alfa Enzyme Replacement Therapy in Patients with Type 1 Gaucher Disease and the Transition From Clinic to Home Infusions During Treatment Protocol HGT-GCB-058

Gregory M Pastores, Barry E Rosenbloom, MD, Neal J Weinreb, Ozlem Goker-Alpan, Rebecca Mardach, Mark Lipson, Jennifer Ibrahim, Gabriel M Cohn, David Zahrieh, Pramod K Mistry

Blood (2011) 118 (21): 1101.

https://doi.org/10.1182/blood.V118.21.1101.1101

Published: 2011

Journal Articles

Improvement in hematological, visceral, and skeletal manifestations of Gaucher disease type 1 with oral eliglustat tartrate (Genz-112638) treatment: 2-year results of a phase 2 study

Elena Lukina, Nora Watman, Elsa Avila Arreguin, Marta Dragosky, Marcelo Iastrebner, Hanna Rosenbaum, Mici Phillips, Gregory M. Pastores, Ravi S. Kamath, Daniel I. Rosenthal, Mathilde Kaper, Tejdip Singh, Ana Cristina Puga, M. Judith Peterschmitt

Blood (2010) 116 (20): 4095–4098.

https://doi.org/10.1182/blood-2010-06-293902

Published: 2010

Journal Articles

A phase 2 study of eliglustat tartrate (Genz-112638), an oral substrate reduction therapy for Gaucher disease type 1

Elena Lukina, Nora Watman, Elsa Avila Arreguin, Maryam Banikazemi, Marta Dragosky, Marcelo Iastrebner, Hanna Rosenbaum, Mici Phillips, Gregory M. Pastores, Daniel I. Rosenthal, Mathilde Kaper, Tejdip Singh, Ana Cristina Puga, Peter L. Bonate, M. Judith Peterschmitt

Blood (2010) 116 (6): 893–899.

https://doi.org/10.1182/blood-2010-03-273151

Published: 2010

NARROW

Type II NKT-T FH cells against Gaucher lipids regulate B-cell immunity and inflammation

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

Type II NKT-TFH Cells Against Gaucher Lipids Regulate B Cell Immunity and Inflammation

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 Month Results

Eliglustat, an Investigational Oral Therapy for Gaucher Disease Type 1: Phase 2 Results After 4 Years of Treatment

Improvements in Skeletal Manifestations in Gaucher Disease Type 1 Patients After 3 Years of Treatment with Oral Eliglustat During a Phase 2 Trial,

Report on the Safety of Velaglucerase Alfa Enzyme Replacement Therapy in Patients with Type 1 Gaucher Disease and the Transition From Clinic to Home Infusions During Treatment Protocol HGT-GCB-058

Improvement in hematological, visceral, and skeletal manifestations of Gaucher disease type 1 with oral eliglustat tartrate (Genz-112638) treatment: 2-year results of a phase 2 study

A phase 2 study of eliglustat tartrate (Genz-112638), an oral substrate reduction therapy for Gaucher disease type 1

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American Society of Hematology

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American Society of Hematology

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