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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

Type 1 Gaucher Disease Severity and Risk for Liver Fibrosis in Untreated Patients and in Patients Treated Primarily with Enzyme Replacement Therapy for a Median of 20 Years

Neal J Weinreb, MD, Robert A Ali, MD

Blood (2018) 132 (Supplement 1): 2403.

https://doi.org/10.1182/blood-2018-99-117215

Published: 2018

Journal Articles

Transformation in Pre-Treatment Presentations of Gaucher Disease during the First Two Decades of Imiglucerase Enzyme Replacement Therapy: A Report from the International Collaborative Gaucher Group Gaucher Registry

Pramod Mistry, MD PhD FRCP, Neal J. Weinreb, MD FACP, Julie Batista, ScM, ScD, Hans C. Andersson, MD, Manisha Balwani, MD MS, Thomas Andrew Burrow, MD, Joel Charrow, MD, Paige Kaplan, MB, BCh, Aneal Khan, MD MSc, FCCMG, FRCPC, Priya S. Kishnani, MD MB BS, Edwin H. Kolodny, MD, Barry E. Rosenbloom, MD, C. Ronald Scott, MD

Blood (2016) 128 (22): 4877.

https://doi.org/10.1182/blood.V128.22.4877.4877

Published: 2016

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

Evaluation of Disease Burden and Response to Treatment in Adults with Type 1 Gaucher Disase Using a Validated DS3 Severity Score Index

Neal J. Weinreb, MD FACP, Eleanor Feingold, PhD, Suma Shankar, MD PhD, Barry E. Rosenbloom, MD, David Finegold, MD, PhD

Blood (2014) 124 (21): 4957.

https://doi.org/10.1182/blood.V124.21.4957.4957

Published: 2014

Journal Articles

Causes of Death in 184 Patients with Type 1 Gaucher Disease From the United States Who Were Never Treated with Enzyme Replacement Therapy

Neal J. Weinreb, MD, Deborah Barbouth, MD, Robert E Lee, MD

Blood (2011) 118 (21): 3128.

https://doi.org/10.1182/blood.V118.21.3128.3128

Published: 2011

Journal Articles

Thrombocytopenia in the Absence of Splenomegaly in Patients with Type 1 Gaucher Disease: A Preliminary Analysis From the ICGG Gaucher Registry,

Barry E Rosenbloom, MD, Sarah Kulke, MD, John Taylor, Neal J. Weinreb, MD

Blood (2011) 118 (21): 4217.

https://doi.org/10.1182/blood.V118.21.4217.4217

Published: 2011

Journal Articles

Determinants of Persisting Thrombocytopenia In Patients with Type 1 Gaucher Disease Treated with Alglucerase/Imiglucerase for 4–5 Years

Maria D. Cappellini, MD, Nadia Belmatoug, Alex Cole, Stephan vom Dahl, Patrick Deegan, Jack Goldblatt, Barry E. Rosenbloom, MD, Anna Tylki-Szymañska, Neal J. Weinreb, MD, Ari Zimran, MD, Carla Hollak, MD, PhD

Blood (2010) 116 (21): 4719.

https://doi.org/10.1182/blood.V116.21.4719.4719

Published: 2010

Journal Articles

Long-Term Data From the ICGG Gaucher Registry: Clinical Parameters After 10 Years of Treatment with Imiglucerase.

Neal J. Weinreb, MD, Stephan vom Dahl, MD, Maria Domenica Cappellini

Blood (2009) 114 (22): 3590.

https://doi.org/10.1182/blood.V114.22.3590.3590

Published: 2009

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

A Report from the International Collaborative Gaucher Group (ICGG) Gaucher Registry

Neal J. Weinreb, MD, Stephan vom Dahl, MD

Blood (2008) 112 (11): 3549.

https://doi.org/10.1182/blood.V112.11.3549.3549

Published: 2008

Journal Articles

Increased Risk of Osteopenic Fractures in Elderly Patients with Gaucher Disease.

Neal J. Weinreb, MD, Lisa Costantini, LPN

Blood (2007) 110 (11): 2403.

https://doi.org/10.1182/blood.V110.11.2403.2403

Published: 2007

Journal Articles

Pharmacological Chaperone Therapy for the Treatment of Gaucher Disease: Results of Phase 1 Clinical Trials and a Clinical Ex Vivo Response Study with a Survey of Blood Markers for 53 Gaucher Patients.

Neal J. Weinreb, MD, G.A. Grabowski, MD, G.M. Pastores, MD, P.M. Fernhoff, MD, P.B. Kaplan, MD, D. Palling, PhD, C.W. Pine, B.E. Raines, F. Insinga, D.J. Lockhart, PhD, H.V. Do, PhD, B.A. Wustman, PhD

Blood (2007) 110 (11): 2404.

https://doi.org/10.1182/blood.V110.11.2404.2404

Published: 2007

Journal Articles

Changing Patterns of Mortality in Gaucher Disease Prior to and Following the Advent of Enzyme Replacement Therapy.

Neal J. Weinreb, MD, Robert E. Lee, MD

Blood (2004) 104 (11): 3799.

https://doi.org/10.1182/blood.V104.11.3799.3799

Published: 2004

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Biomarker Response to Oral Eliglustat in Adults with Gaucher Disease Type 1: Results from 4 Completed Clinical Trials

Type 1 Gaucher Disease Severity and Risk for Liver Fibrosis in Untreated Patients and in Patients Treated Primarily with Enzyme Replacement Therapy for a Median of 20 Years

Transformation in Pre-Treatment Presentations of Gaucher Disease during the First Two Decades of Imiglucerase Enzyme Replacement Therapy: A Report from the International Collaborative Gaucher Group Gaucher Registry

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”

Evaluation of Disease Burden and Response to Treatment in Adults with Type 1 Gaucher Disase Using a Validated DS3 Severity Score Index

Causes of Death in 184 Patients with Type 1 Gaucher Disease From the United States Who Were Never Treated with Enzyme Replacement Therapy

Thrombocytopenia in the Absence of Splenomegaly in Patients with Type 1 Gaucher Disease: A Preliminary Analysis From the ICGG Gaucher Registry,

Determinants of Persisting Thrombocytopenia In Patients with Type 1 Gaucher Disease Treated with Alglucerase/Imiglucerase for 4–5 Years

Long-Term Data From the ICGG Gaucher Registry: Clinical Parameters After 10 Years of Treatment with Imiglucerase.

Avascular Necrosis in Untreated Patients with Type 1 Gaucher Disease.

A Report from the International Collaborative Gaucher Group (ICGG) Gaucher Registry

Increased Risk of Osteopenic Fractures in Elderly Patients with Gaucher Disease.

Pharmacological Chaperone Therapy for the Treatment of Gaucher Disease: Results of Phase 1 Clinical Trials and a Clinical Ex Vivo Response Study with a Survey of Blood Markers for 53 Gaucher Patients.

Changing Patterns of Mortality in Gaucher Disease Prior to and Following the Advent of Enzyme Replacement Therapy.

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