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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

Encore - a Randomized, Controlled, Open-Label Non-Inferiority Study Comparing Eliglustat to Imiglucerase in Gaucher Disease Type 1 Patients Stabilized on Enzyme Replacement Therapy: 24-Month Results

Barry E. Rosenbloom, MD, Timothy M. Cox, MD, Guillermo I. Drelichman, MD, Renata Cravo, MD, Manisha Balwani, MD MS, Thomas Andrew Burrow, MD FAAP, FACMG, Ana Maria Martins, MD PhD, Elena Lukina, MD PhD, Leorah H. Ross, MD PhD, Jennifer Angell, ScM, Judith Peterschmitt, MD

Blood (2014) 124 (21): 1406.

https://doi.org/10.1182/blood.V124.21.1406.1406

Published: 2014

Journal Articles

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 Reached Therapeutic Goals

Thomas Andrew Burrow, MD, FAAP, FACMG, Manisha Balwani, MD, MS, Timothy M. Cox, MD, Guillermo Drelichman, MD, Renata Cravo, MD, Ana Maria Martins, MD, PhD, Elena Lukina, MD, PhD, Barry E. Rosenbloom, MD, Leorah H. Ross, MD, PhD, Jennifer Angell, ScM, Ana Cristina Puga, MD, PhD

Blood (2013) 122 (21): 3468.

https://doi.org/10.1182/blood.V122.21.3468.3468

Published: 2013

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

Eliglustat, An Investigational Oral Therapy for Gaucher Disease Type 1: Phase 2 Results After 3 Years

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

Blood (2011) 118 (21): 1104.

https://doi.org/10.1182/blood.V118.21.1104.1104

Published: 2011

NARROW

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

Encore - a Randomized, Controlled, Open-Label Non-Inferiority Study Comparing Eliglustat to Imiglucerase in Gaucher Disease Type 1 Patients Stabilized on Enzyme Replacement Therapy: 24-Month Results

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 Reached Therapeutic Goals

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,

Eliglustat, An Investigational Oral Therapy for Gaucher Disease Type 1: Phase 2 Results After 3 Years

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ASH Publications

American Society of Hematology

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

Encore - a Randomized, Controlled, Open-Label Non-Inferiority Study Comparing Eliglustat to Imiglucerase in Gaucher Disease Type 1 Patients Stabilized on Enzyme Replacement Therapy: 24-Month Results

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 Reached Therapeutic Goals

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,

Eliglustat, An Investigational Oral Therapy for Gaucher Disease Type 1: Phase 2 Results After 3 Years

Email alerts

ASH Publications

American Society of Hematology

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