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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

Bone Marrow Involvement in Patients with Type 1 Gaucher Disease Receiving Low-Dose Long Term Enzyme Replacement Therapy (median 18 years)

Shoshana Revel-Vilk, MD, Jeffrey Szer, MBBS, Michal Becker Cohen, Ari Zimran, MD

Blood (2019) 134 (Supplement_1): 4862.

https://doi.org/10.1182/blood-2019-131013

Published: 2019

Journal Articles

Liver-Directed AAV Gene Therapy for Gaucher Disease

Carlos J Miranda, PhD, Miriam Canavese, PhD, Elisa Chisari, MS, Jalpa Pandya, MPhil, Clement Cocita, PhD, Maria Portillo, MRes, Jenny McIntosh, PhD, Azadeh Kia, PhD, Jonathan H. Foley, PhD, Allison Dane, PhD, Jey M Jeyakumar, PhD, Rose Sheridan, PhD, Romuald Corbau, Amit C. Nathwani, MD PhD

Blood (2019) 134 (Supplement_1): 3354.

https://doi.org/10.1182/blood-2019-124280

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

Early Diagnosis of an Infant with Gaucher's Disease Type 3. Case Report

Adriana C. Bello, MD, Rossana Cortez

Blood (2018) 132 (Supplement 1): 4950.

https://doi.org/10.1182/blood-2018-99-110263

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

In Vitro Erythropoietic Response to Enzyme Replacement Therapy in Gaucher Type 1 Patients

Paola Delbini, PhD, Irene Motta, Elena Cassinerio, Lorena Duca, Elena Trombetta, Margherita Migone De Amicis, Maria Domenica Cappellini, MD

Blood (2017) 130 (Supplement 1): 3476.

https://doi.org/10.1182/blood.V130.Suppl_1.3476.3476

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

An Unsuspected Dyserythropoiesis in Gaucher Disease

Niloofar Reihani, PhD, Jean-Benoît Arlet, MD PhD, Thierry Billette de Villemeur, MD PhD, Nadia Belmatoug, MD, Yves Colin, PhD, Olivier Hermine, MD PhD, Caroline Le Van Kim, PhD, Mélanie Franco, PhD

Blood (2014) 124 (21): 1344.

https://doi.org/10.1182/blood.V124.21.1344.1344

Published: 2014

Journal Articles

Novel Management of Immune Thrombocytopenia in Gaucher Disease Patients

Hanna Rosenbaum, MD

Blood (2014) 124 (21): 5006.

https://doi.org/10.1182/blood.V124.21.5006.5006

Published: 2014

Journal Articles

Influence Of Genetic Variability Related To Bone Remodeling and Cytokine Profile In The Development Of Bone Involvement In Gaucher Disease

Javier Gervas-Arruga, PhD, Jorge Cebolla, Ignacio de Blas, Mercedes Roca, MD PhD, Miguel Pocovi, Prof, Pilar Giraldo, MD PhD

Blood (2013) 122 (21): 3469.

https://doi.org/10.1182/blood.V122.21.3469.3469

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

Gaucher Disease and Multiple Myeloma: A Diagnostic and Treatment Challenge

Hanna Rosenbaum, MD, Irit Avivi, MD, Eliezer A. Rachmilewitz, Paulina Petchenko, MD

Blood (2011) 118 (21): 2166.

https://doi.org/10.1182/blood.V118.21.2166.2166

Published: 2011

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

Gaucher Disease and LGL Proliferations: Provocative Commonality?

Rosa Ruchlemer, MD, Deborah Elstein, PhD, Eti Broide, Constantine Reinus, Hannah Maayaan, Ari Zimran, MD

Blood (2011) 118 (21): 1100.

https://doi.org/10.1182/blood.V118.21.1100.1100

Published: 2011

Journal Articles

The Uptake of Recombinant Glucocerebrosidases by Blood Monocytes From Type 1 Gaucher Disease Patients Is Heterogeneous

Juliette Berger, Jérome Stirnemann, MD, PhD, Céline Bourgne, Bruno Pereira, Pascale Pigeon, Djazia Heraoui, Chantal Rapatel, Christian Rose, MD, PhD, Nadia Belmatoug, MD, Marc G Berger, MD, PhD

Blood (2011) 118 (21): 1102.

https://doi.org/10.1182/blood.V118.21.1102.1102

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

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

Immune Thrombocytopenia in Type I Gaucher Disease.

Hanna Rosenbaum, MD, Tina Napso, MS.c, Lilach Bonstein, Ph.D

Blood (2007) 110 (11): 3200.

https://doi.org/10.1182/blood.V110.11.3200.3200

Published: 2007

NARROW

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

Bone Marrow Involvement in Patients with Type 1 Gaucher Disease Receiving Low-Dose Long Term Enzyme Replacement Therapy (median 18 years)

Liver-Directed AAV Gene Therapy for Gaucher Disease

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

Early Diagnosis of an Infant with Gaucher's Disease Type 3. Case Report

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

In Vitro Erythropoietic Response to Enzyme Replacement Therapy in Gaucher Type 1 Patients

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

An Unsuspected Dyserythropoiesis in Gaucher Disease

Novel Management of Immune Thrombocytopenia in Gaucher Disease Patients

Influence Of Genetic Variability Related To Bone Remodeling and Cytokine Profile In The Development Of Bone Involvement In Gaucher Disease

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

Gaucher Disease and Multiple Myeloma: A Diagnostic and Treatment Challenge

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

Gaucher Disease and LGL Proliferations: Provocative Commonality?

The Uptake of Recombinant Glucocerebrosidases by Blood Monocytes From Type 1 Gaucher Disease Patients Is Heterogeneous

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.

Immune Thrombocytopenia in Type I Gaucher Disease.

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American Society of Hematology

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American Society of Hematology

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