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Journal Articles

Largest Cohort Study of Gaucher Disease Type 3 from a Single Center in Egypt Spanning Two Decades

Amal El-Beshlawy, MD, Khaled Mohamed Abd El-Azim Eid, MD, Amina AbdelSalam, MD, Fadwa Said Mohamed, MD, Yasmeen M.M. Selim, Nadeen Gebril, Ekram Fateen, Pramod K. Mistry, MD PhD FRCP

Blood (2020) 136 (Supplement 1): 21–22.

https://doi.org/10.1182/blood-2020-141776

Published: 2020

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

MGUS, lymphoplasmacytic malignancies, and Gaucher disease: the significance of the clinical association

Neal J. Weinreb, Pramod K. Mistry, Barry E. Rosenbloom, Madhav V. Dhodapkar

Blood (2018) 131 (22): 2500–2501.

https://doi.org/10.1182/blood-2018-02-834689

Published: 2018

Journal Articles

Type II NKT-T FH cells against Gaucher lipids regulate B-cell immunity and inflammation

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

Blood (2015) 125 (8): 1256–1271.

https://doi.org/10.1182/blood-2014-09-600270

Published: 2015

Includes: Supplemental data

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

Phenotype diversity in type 1 Gaucher disease: discovering the genetic basis of Gaucher disease/hematologic malignancy phenotype by individual genome analysis

Sarah M. Lo, Murim Choi, Jun Liu, Dhanpat Jain, Rolf G. Boot, Wouter W. Kallemeijn, Johannes M. F. G. Aerts, Farzana Pashankar, Gary M. Kupfer, Shrikant Mane, Richard P. Lifton, Pramod K. Mistry

Blood (2012) 119 (20): 4731–4740.

https://doi.org/10.1182/blood-2011-10-386862

Published: 2012

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

Elevated plasma glucosylsphingosine in Gaucher disease: relation to phenotype, storage cell markers, and therapeutic response

Nick Dekker, Laura van Dussen, Carla E. M. Hollak, Herman Overkleeft, Saskia Scheij, Karen Ghauharali, Mariëlle J. van Breemen, Maria J. Ferraz, Johanna E. M. Groener, Mario Maas, Frits A. Wijburg, Dave Speijer, Anna Tylki-Szymanska, Pramod K. Mistry, Rolf G. Boot, Johannes M. Aerts

Blood (2011) 118 (16): e118–e127.

https://doi.org/10.1182/blood-2011-05-352971

Published: 2011

Includes: Supplemental data

Journal Articles

Avascular Necrosis in Untreated Patients with Type 1 Gaucher Disease.

Pramod K Mistry, MD, PhD, FRCP, Patrick Deegan, MD, Ashok Vellodi, MD, J. Alexander Cole, DSc, MPH, Michael Yeh, MD, Neal J. Weinreb, MD

Blood (2009) 114 (22): 1353.

https://doi.org/10.1182/blood.V114.22.1353.1353

Published: 2009

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

Consequences of Diagnostic Delays in Type 1 Gaucher Disease: A Unique Opportunity among Hematologists/Oncologists for Early Diagnosis and Intervention.

Pramod K. Mistry, MD, PhD, FRCP, Sara Sadan, MD, Ruhua Yang, MD, John Yee, MD, Mei Yang, MD

Blood (2006) 108 (11): 3308.

https://doi.org/10.1182/blood.V108.11.3308.3308

Published: 2006

NARROW

Largest Cohort Study of Gaucher Disease Type 3 from a Single Center in Egypt Spanning Two Decades

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

MGUS, lymphoplasmacytic malignancies, and Gaucher disease: the significance of the clinical association

Type II NKT-T FH cells against Gaucher lipids regulate B-cell immunity and inflammation

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

Phenotype diversity in type 1 Gaucher disease: discovering the genetic basis of Gaucher disease/hematologic malignancy phenotype by individual genome analysis

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

Elevated plasma glucosylsphingosine in Gaucher disease: relation to phenotype, storage cell markers, and therapeutic response

Avascular Necrosis in Untreated Patients with Type 1 Gaucher Disease.

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

Consequences of Diagnostic Delays in Type 1 Gaucher Disease: A Unique Opportunity among Hematologists/Oncologists for Early Diagnosis and Intervention.

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American Society of Hematology

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ASH Publications

American Society of Hematology

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