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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
Journal: Blood
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
Journal: Blood
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
Journal: Blood
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

Clinical Trials & Observations
Neal J. Weinreb, Pramod K. Mistry, Barry E. Rosenbloom, Madhav V. Dhodapkar
Journal: Blood
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
Journal: Blood
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
Journal: Blood
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
Journal: Blood
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
Journal: Blood
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

e-blood
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
Journal: Blood
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
Journal: Blood
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
Journal: Blood
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
Journal: Blood
Blood (2006) 108 (11): 3308.
https://doi.org/10.1182/blood.V108.11.3308.3308
Published: 2006