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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

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

Iron Overload in Patients with Paroxysmal Nocturnal Hemoglobinuria

Kira Lukina, MD PhD, Nina Tsvetaeva, MD PhD, Olga Nikoulina, MD PhD, Elena Sysoeva, MD PhD, Valentin Sinitsyn, MD PhD Professor, Elena Lukina, MD PhD, Valery G. Savchenko, MD PhD academician

Blood (2018) 132 (Supplement 1): 1051.

https://doi.org/10.1182/blood-2018-99-114654

Published: 2018

Journal Articles

Prospective National-Base Multicenter Study to Standardize the Follow-up of Type 1Gaucher Disease Patients Treated with Eliglustat Under Standard of Care Practice. Trazelga Project

Marcio M Andrade-Campos, PhD, Jorge J Cebolla, MSc, Laura Lopez de Frutos, PhD, Pilar Irun, PhD, Soledad Noya, MD, Santiago Nieto, MD, Lucia Villalon, PhD, Ana I Arribas, MD, Abelardo Barez, PhD, Javier Garcia-Frade, PD, Maria Lourdes Hermosin, MD, Jesús-María Hernández-Rivas, MD PhD, Daiana Ibarretxe, MD, Maria L Lozano, MD PhD, Teresa Molero, PhD, Maria Angeles Perez-Saenz, MDPhD, Jesus Villarrubia, PhD, Pilar Giraldo, PhD

Blood (2018) 132 (Supplement 1): 4942.

https://doi.org/10.1182/blood-2018-99-115534

Published: 2018

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 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

Eliglustat maintains long-term clinical stability in patients with Gaucher disease type 1 stabilized on enzyme therapy

Timothy M. Cox, Guillermo Drelichman, Renata Cravo, Manisha Balwani, Thomas Andrew Burrow, Ana Maria Martins, Elena Lukina, Barry Rosenbloom, Ozlem Goker-Alpan, Nora Watman, Amal El-Beshlawy, Priya S. Kishnani, Maria Lucia Pedroso, Sebastiaan J. M. Gaemers, Regina Tayag, M. Judith Peterschmitt

Blood (2017) 129 (17): 2375–2383.

https://doi.org/10.1182/blood-2016-12-758409

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

Long Term Outcomes of ERT in Children with Gaucher Disease. Spanish Experience

Marcio M Andrade, MD, Pilar Alfonso, PhD, Pilar Irún, PhD, Javier Dalmau, MD, Jose Luis Barbera, MD, Horacio Cano, MD, María Angeles Fernandez-Galan, MD, Rafael Franco, MD, Inmaculada Garcia, MD, Angela IbaÃ±ez, MD, Francisco Lendinez, MD, Elena Martin-Hernandez, MD, Luis Antonio Sancho-Val, MD, Pablo Sanjurjo, MDPhD, Miguel Pocovi, Prof, Pilar Giraldo, MD PhD

Blood (2014) 124 (21): 4950.

https://doi.org/10.1182/blood.V124.21.4950.4950

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

Journal Articles

Lukina E, Watman N, Arreguin EA, et al. 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. Blood . 2010;116(20): 4095–4098 .

Blood (2011) 117 (20): 5551.

https://doi.org/10.1182/blood-2011-04-347146

Published: 2011

Journal Articles

Eliglustat Tartrate, a Novel Investigational Oral Substrate Reduction Therapy for Gaucher Disease Type 1: Updated Phase 2 Results.

Elena Lukina, Judith Peterschmitt, Nora Watman, Marta Dragosky, Marcelo Iastrebner, Gregory Pastores, Elsa Avila Arreguin, Hanna Rosenbaum, Mici Phillips, Mathilde Kaper, Arshadul Haque, Ana Cristina Puga

Blood (2010) 116 (21): 3791.

https://doi.org/10.1182/blood.V116.21.3791.3791

Published: 2010

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

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

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

Iron Overload in Patients with Paroxysmal Nocturnal Hemoglobinuria

Prospective National-Base Multicenter Study to Standardize the Follow-up of Type 1Gaucher Disease Patients Treated with Eliglustat Under Standard of Care Practice. Trazelga Project

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 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

Eliglustat maintains long-term clinical stability in patients with Gaucher disease type 1 stabilized on enzyme therapy

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

Long Term Outcomes of ERT in Children with Gaucher Disease. Spanish Experience

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

Lukina E, Watman N, Arreguin EA, et al. 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. Blood . 2010;116(20): 4095–4098 .

Eliglustat Tartrate, a Novel Investigational Oral Substrate Reduction Therapy for Gaucher Disease Type 1: Updated Phase 2 Results.

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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