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Journal Articles

Bone Marrow Plasma Cell Burden in Gaucher Disease Correlates with Local Macrophage Infiltration and May be Altered By Disease Treatment

Monika Klimkowska, MD PhD, Maciej J. Machaczka, MD PhD

Blood (2019) 134 (Supplement_1): 3593.

https://doi.org/10.1182/blood-2019-131343

Published: 2019

Journal Articles

Not All Patients with Mild Phenotype of Gaucher Disease (GD) Need Disease Specific Treatment As Reflected By Patients Reported Outcomes Measures

Shoshana Revel-Vilk, MD, Tama Dinur, PhD, Majdolen Istaiti, Dafna Frydman, DMV, Michal Becker-Cohen, MSc, Ari Zimran, MD

Blood (2019) 134 (Supplement_1): 4712.

https://doi.org/10.1182/blood-2019-123000

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

Twenty-Five Years Diagnosing Gaucher’s Disease in Spain, What We Have Learned?

Marcio M Andrade-Campos, PhD, Jose L Capablo, PhD, Jesus J Fraile, PhD, Mercedes Roca-Espiau, PhD, Pilar Irun, PhD, Jorge J Cebolla, MSc, Laura Lopez de Frutos, PhD, Izaskun Arenaz, MSci, Jesus Villarrubia, PhD, Abelardo Barez, PhD, Horacio Cano, MD, Teresa Molero, PhD, Lucia Villalon, PhD, Pilar Giraldo, PhD

Blood (2018) 132 (Supplement 1): 1110.

https://doi.org/10.1182/blood-2018-99-115474

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

Assessment of Hematologic and Visceral Parameters in Patients with Type 1 Gaucher Disease in Spain: An Epidemiological Study Using the Therapeutic Goals MAP (Monitor, Action and Progress) Tool ®

Pilar Giraldo, Jordi Perez, Ramiro Núñez, Rafael Fernandez de la Puebla, Elisa Luño, Salvador Saura, Juan Carlos Bureo, Javier de la Serna, Sylvia Plaza, Víctor Navas

Blood (2014) 124 (21): 4963.

https://doi.org/10.1182/blood.V124.21.4963.4963

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

Substrate Reduction Therapy With Miglustat In Type 1 Gaucher Disease In Spain. Nine Years Outcomes Update On ZAGAL Study

Pilar Giraldo, MD PhD, Marcio M Andrade, Sr., MD, Blanca Medrano, Pilar Alfonso, PhD, Pilar Irún, PhD, Koldo Atutxa, MD, Angeles Fernandez-Galan, MD, Abelardo Barez, MD, Rafael Franco, MD, Inmaculada Roig, Vicente Giner, MD, Lucia Villalon, MD, Elisa Martinez-Estefano, MD, Elisa Luño, MD, Ines Loyola, MD, Olga Salamero, Javier de la Serna, MD, PhD, Miguel Pocovi

Blood (2013) 122 (21): 4713.

https://doi.org/10.1182/blood.V122.21.4713.4713

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

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

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

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

Journal Articles

Changes in the Atherogenic Profile in Type 1 Gaucher Disease Patients after Substrate Reduction Therapy

Pilar Giraldo, MD, PhD, Jose Puzo, Pilar Alfonso, Pilar Irun, Miguel Pocovi

Blood (2008) 112 (11): 4645.

https://doi.org/10.1182/blood.V112.11.4645.4645

Published: 2008

Journal Articles

Diagnostic and Disease Management Algorithms for Gaucher Disease: A Guide for Haematologists

Pramod K Mistry, Maria Domenica Cappellini, Elena Lukina, Hayri Özsan, Sara Mach Pascual, Hanna Rosenbaum, Maria Helena Solano, Zachary Simon Spigelman, Jesús Villarrubia, Nora Patricia Watman, Gero Massenkeil

Blood (2008) 112 (11): 4648.

https://doi.org/10.1182/blood.V112.11.4648.4648

Published: 2008

Journal Articles

Gaucher Disease: A Systematic Review and Meta-Analysis of Bone Complications and Their Response to Treatment.

Siavash Piran, MSc, Dominick Amato, MD

Blood (2008) 112 (11): 1265.

https://doi.org/10.1182/blood.V112.11.1265.1265

Published: 2008

Journal Articles

Clinical Evaluation of CCL18/PARC and Chitotriosidase Biomarkers in Gaucher Disease.

Pilar Alfonso, MD, PhD, Paz Latre, MD, Ignacio de Blas, MDPhD, Pilar Giraldo, MDPhD, Manuel Giralt, MDPhD, Miguel Pocovi, MDPhD

Blood (2005) 106 (11): 3885.

https://doi.org/10.1182/blood.V106.11.3885.3885

Published: 2005

Journal Articles

Allogeneic BMT Followed by Substrate Deprivation Therapy with N-butyldeoxynojirimycin in a Child with Tay-Sachs Disease.

Hans J.F.M. Jacobs, Michel M.A.A.P. Willemsen, Ph.D., M.D., Jacqueline J.J. Groot-Loonen, Ph.D. M.D., Ron R.A. Wevers, Ph.D. M.D., Peter P.M. Hoogerbrugge, Ph.D. M.D.

Blood (2004) 104 (11): 5158.

https://doi.org/10.1182/blood.V104.11.5158.5158

Published: 2004

NARROW

Bone Marrow Plasma Cell Burden in Gaucher Disease Correlates with Local Macrophage Infiltration and May be Altered By Disease Treatment

Not All Patients with Mild Phenotype of Gaucher Disease (GD) Need Disease Specific Treatment As Reflected By Patients Reported Outcomes Measures

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

Twenty-Five Years Diagnosing Gaucher’s Disease in Spain, What We Have Learned?

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

Assessment of Hematologic and Visceral Parameters in Patients with Type 1 Gaucher Disease in Spain: An Epidemiological Study Using the Therapeutic Goals MAP (Monitor, Action and Progress) Tool ®

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

Substrate Reduction Therapy With Miglustat In Type 1 Gaucher Disease In Spain. Nine Years Outcomes Update On ZAGAL Study

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

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

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

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

Changes in the Atherogenic Profile in Type 1 Gaucher Disease Patients after Substrate Reduction Therapy

Diagnostic and Disease Management Algorithms for Gaucher Disease: A Guide for Haematologists

Gaucher Disease: A Systematic Review and Meta-Analysis of Bone Complications and Their Response to Treatment.

Clinical Evaluation of CCL18/PARC and Chitotriosidase Biomarkers in Gaucher Disease.

Allogeneic BMT Followed by Substrate Deprivation Therapy with N-butyldeoxynojirimycin in a Child with Tay-Sachs Disease.

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American Society of Hematology

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American Society of Hematology

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