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Journal Articles

Evaluation of in Vitro Macrophage Characterization in Gaucher Type 1 (GD1) Patients

Paola Delbini, PhD, Viola Ghiandai, Maria Domenica Cappellini, MD, Lorena Duca, Isabella Nava, Elena Cassinerio, MD, Irene Motta, MD

Blood (2019) 134 (Supplement_1): 3592.

https://doi.org/10.1182/blood-2019-130032

Published: 2019

Journal Articles

Preliminary Results of a Prospective Observational Study to Assess the Prevalence of Gaucher Disease in an Adult Population Affected By MGUS

Gaetano Giuffrida, MD, Concetta Conticello, MD, Daniela Nicolosi, PharmD, Valeria Calafiore, Alessandra Romano, MD PhD, Ugo Consoli, MD, Caterina Musolino, MD, Vanessa Innao, Flavia Fiorenza, MD, Cinzia Maugeri, MD, Giuseppe Longo, MD, Maria Rocca Cingari, MD, Francesco Di Raimondo, MD

Blood (2019) 134 (Supplement_1): 4868.

https://doi.org/10.1182/blood-2019-129944

Published: 2019

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

Identification of GBA Gene Mutations in 19 Pakistani Unrelated Patients of Gaucher Disease

Arshi Naz, PhD, Qurat Abedin, M.Sc, Shariq Ahmed, MPhil, Saima Siddiqui, FCPS, Tahir Shamsi

Blood (2018) 132 (Supplement 1): 4952.

https://doi.org/10.1182/blood-2018-99-120385

Published: 2018

Journal Articles

Altering Glycosphingolipid Composition to Improve Multiple Myeloma Bone Complication

Houfu Leng, MSc, DPhil, MBBS, BSc, Adel Ersek, PhD, Emma Morris, DPhil, Beatriz Gamez Molina, PhD, Claire M. Edwards, PhD, Nicole Horwood, PhD

Blood (2018) 132 (Supplement 1): 1942.

https://doi.org/10.1182/blood-2018-99-112237

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

Animal Models of Aging Research

Marc F. Hoylaerts, PhD

Blood (2015) 126 (23): SCI-4.

https://doi.org/10.1182/blood.V126.23.SCI-4.SCI-4

Published: 2015

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

“Imiglucerase Treatment Associated with Reduction of Bone Claims in Gaucher Patients: Analysis of US Claims Data”

Lubomyra Nalysnyk, MD MPH, Martin Selzer, PhD, Alaa Hamed, MD MPH, MBA, Neal J. Weinreb, MD FACP

Blood (2014) 124 (21): 4837.

https://doi.org/10.1182/blood.V124.21.4837.4837

Published: 2014

Journal Articles

Type II NKT-TFH Cells Against Gaucher Lipids Regulate B Cell Immunity and Inflammation

Shiny Nair, PhD, Chandra Sekhar Boddupalli, PhD, Rakesh Verma, PhD, Jun Liu, Yang Ruhua, Gregory M Pastores, MD, Pramod Mistry, MD PhD FRCP, Madhav V. Dhodapkar, MD

Blood (2014) 124 (21): 755.

https://doi.org/10.1182/blood.V124.21.755.755

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

New Derivatives of L-Idonojirimycin Working As Pharmacological Chaperones for the Treatment of Gaucher Disease

Pilar Alfonso, PhD, Javier Gervas, Vanesa Andreu, PhD, Joaquin Navascues, PhD, Francisca Sanchez-Jimenez, Prof, Miguel Pocovi, Prof, Carmen Ortiz-Mellet, Prof, Jose M Garcia-Fernandez, Prof, Pilar Giraldo, MD, PhD

Blood (2012) 120 (21): 3270.

https://doi.org/10.1182/blood.V120.21.3270.3270

Published: 2012

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

Report on the Safety of Velaglucerase Alfa Enzyme Replacement Therapy in Patients with Type 1 Gaucher Disease and the Transition From Clinic to Home Infusions During Treatment Protocol HGT-GCB-058

Gregory M Pastores, Barry E Rosenbloom, MD, Neal J Weinreb, Ozlem Goker-Alpan, Rebecca Mardach, Mark Lipson, Jennifer Ibrahim, Gabriel M Cohn, David Zahrieh, Pramod K Mistry

Blood (2011) 118 (21): 1101.

https://doi.org/10.1182/blood.V118.21.1101.1101

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

Evaluation of in Vitro Macrophage Characterization in Gaucher Type 1 (GD1) Patients

Preliminary Results of a Prospective Observational Study to Assess the Prevalence of Gaucher Disease in an Adult Population Affected By MGUS

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

Identification of GBA Gene Mutations in 19 Pakistani Unrelated Patients of Gaucher Disease

Altering Glycosphingolipid Composition to Improve Multiple Myeloma Bone Complication

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

Animal Models of Aging Research

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

“Imiglucerase Treatment Associated with Reduction of Bone Claims in Gaucher Patients: Analysis of US Claims Data”

Type II NKT-TFH Cells Against Gaucher Lipids Regulate B Cell Immunity and Inflammation

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

New Derivatives of L-Idonojirimycin Working As Pharmacological Chaperones for the Treatment of Gaucher Disease

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,

Report on the Safety of Velaglucerase Alfa Enzyme Replacement Therapy in Patients with Type 1 Gaucher Disease and the Transition From Clinic to Home Infusions During Treatment Protocol HGT-GCB-058

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

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American Society of Hematology

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ASH Publications

American Society of Hematology

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