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Journal Articles

Biomarker Response to Oral Eliglustat in Adults with Gaucher Disease Type 1: Results from 4 Completed Clinical Trials

Neal J Weinreb, MD, Timothy M Cox, MD, Pramod K. Mistry, MD PhD FRCP, Joel Charrow, MD, Elena Lukina, MD PhD Professor, Meredith C. Foster, ScD, M. Judith Peterschmitt, MD

Blood (2019) 134 (Supplement_1): 4859.

https://doi.org/10.1182/blood-2019-127337

Published: 2019

Journal Articles

The Long-Term Adverse Event Profile of Oral Eliglustat for the Treatment of Gaucher Disease Type 1: Pooled Analysis of Data from 393 Patients in 4 Completed Trials

M. Judith Peterschmitt, MD, Selena Freisens, MD, Audrey W Hou, PharmD, Lisa Underhill, MS, Meredith C. Foster, ScD, Sebastiaan J.M. Gaemers, MD

Blood (2018) 132 (Supplement 1): 3692.

https://doi.org/10.1182/blood-2018-99-118319

Published: 2018

Journal Articles

Long-Term Effects of Oral Eliglustat on Skeletal Manifestations of Gaucher Disease Type 1: Results from Four Completed Clinical Trials

Pramod K. Mistry, MD PhD FRCP, Joel Charrow, MD, Timothy Cox, MD, Elena Lukina, MD PhD, Theodore Marinakis, MD, Meredith C. Foster, ScD, Sebastiaan J.M. Gaemers, MD, Judith Peterschmitt, MD

Blood (2018) 132 (Supplement 1): 2396.

https://doi.org/10.1182/blood-2018-99-117289

Published: 2018

Journal Articles

Long-Term Hematologic Response to Oral Eliglustat in Treatment-Naive Patients with Gaucher Disease Type 1: Final Results from a Phase 2 and the Phase 3 Engage Trials

Heather Lau, MD, Elena Lukina, MD, Pramod Mistry, MD, Yaoshi Wu, Sebastiaan J.M. Gaemers, MD, M. Judith Peterschmitt, MD

Blood (2017) 130 (Supplement 1): 448.

https://doi.org/10.1182/blood.V130.Suppl_1.448.448

Published: 2017

Journal Articles

Long-Term Hematologic Response to Eliglustat in Patients with Gaucher Disease Type 1: Results from a Phase 2 and Two Phase 3 Trials

Neal J. Weinreb, MD FACP, Timothy M Cox, MD, Elena Lukina, MD, Pramod Mistry, MD PhD FRCP, Jennifer Angell, Sebastiaan Gaemers, MD, M. Judith Peterschmitt, MD

Blood (2015) 126 (23): 884.

https://doi.org/10.1182/blood.V126.23.884.884

Published: 2015

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

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

Journal Articles

Genz-112638 for Gaucher Disease Type 1: Phase 2 Clinical Trial Results After 18 Months of Treatment.

Judith Peterschmitt, MD, Elena Lukina, PhD, MD, Nora Patricia Watman, MD, Elsa Avila Arreguin, MD, Maryam Banikazemi, MD, Gregory Pastores, MD, Marcelo Iastrebner, MD, Marta Dragosky, MD, Hanna Rosenbaum, MD, Mici Phillips, MD, Mathilde Kaper, Tejdip Singh, MD, Ana Cristina Puga, MD, PhD

Blood (2009) 114 (22): 1349.

https://doi.org/10.1182/blood.V114.22.1349.1349

Published: 2009

NARROW

Biomarker Response to Oral Eliglustat in Adults with Gaucher Disease Type 1: Results from 4 Completed Clinical Trials

The Long-Term Adverse Event Profile of Oral Eliglustat for the Treatment of Gaucher Disease Type 1: Pooled Analysis of Data from 393 Patients in 4 Completed Trials

Long-Term Effects of Oral Eliglustat on Skeletal Manifestations of Gaucher Disease Type 1: Results from Four Completed Clinical Trials

Long-Term Hematologic Response to Oral Eliglustat in Treatment-Naive Patients with Gaucher Disease Type 1: Final Results from a Phase 2 and the Phase 3 Engage Trials

Long-Term Hematologic Response to Eliglustat in Patients with Gaucher Disease Type 1: Results from a Phase 2 and Two Phase 3 Trials

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

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

Genz-112638 for Gaucher Disease Type 1: Phase 2 Clinical Trial Results After 18 Months of Treatment.

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American Society of Hematology

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American Society of Hematology

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