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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

Eliglustat maintains long-term clinical stability in patients with Gaucher disease type 1 stabilized on enzyme therapy

Timothy M. Cox, Guillermo Drelichman, Renata Cravo, Manisha Balwani, Thomas Andrew Burrow, Ana Maria Martins, Elena Lukina, Barry Rosenbloom, Ozlem Goker-Alpan, Nora Watman, Amal El-Beshlawy, Priya S. Kishnani, Maria Lucia Pedroso, Sebastiaan J. M. Gaemers, Regina Tayag, M. Judith Peterschmitt

Blood (2017) 129 (17): 2375–2383.

https://doi.org/10.1182/blood-2016-12-758409

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

Encore - a Randomized, Controlled, Open-Label Non-Inferiority Study Comparing Eliglustat to Imiglucerase in Gaucher Disease Type 1 Patients Stabilized on Enzyme Replacement Therapy: 24-Month Results

Barry E. Rosenbloom, MD, Timothy M. Cox, MD, Guillermo I. Drelichman, MD, Renata Cravo, MD, Manisha Balwani, MD MS, Thomas Andrew Burrow, MD FAAP, FACMG, Ana Maria Martins, MD PhD, Elena Lukina, MD PhD, Leorah H. Ross, MD PhD, Jennifer Angell, ScM, Judith Peterschmitt, MD

Blood (2014) 124 (21): 1406.

https://doi.org/10.1182/blood.V124.21.1406.1406

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

Gaucher Disease Psychosine Mis-Traffics Endocytosed Cargo to a Non-Functional, Pre-Lysosomal Compartment.

Jonathan C. Roos, MA, Susan Z. Wang, MD, Timothy M. Cox, MD

Blood (2006) 108 (11): 1270.

https://doi.org/10.1182/blood.V108.11.1270.1270

Published: 2006

Journal Articles

Spectrum of hemojuvelin gene mutations in 1q-linked juvenile hemochromatosis

Carmela Lanzara, Antonella Roetto, Filomena Daraio, Silvain Rivard, Romina Ficarella, Hervey Simard, Timothy M. Cox, Mario Cazzola, Alberto Piperno, Anne-Paule Gimenez-Roqueplo, Paola Grammatico, Stefano Volinia, Paolo Gasparini, Clara Camaschella

Blood (2004) 103 (11): 4317–4321.

https://doi.org/10.1182/blood-2004-01-0192

Published: 2004

Journal Articles

Screening hepcidin for mutations in juvenile hemochromatosis: identification of a new mutation (C70R)

Antonella Roetto, Filomena Daraio, Paolo Porporato, Roberta Caruso, Timothy M. Cox, Mario Cazzola, Paolo Gasparini, Alberto Piperno, Clara Camaschella

Blood (2004) 103 (6): 2407–2409.

https://doi.org/10.1182/blood-2003-10-3390

Published: 2004

Journal Articles

Marked elevation of the chemokine CCL18/PARC in Gaucher disease: a novel surrogate marker for assessing therapeutic intervention

Rolf G. Boot, Marri Verhoek, Maaike de Fost, Carla E. M. Hollak, Mario Maas, Boris Bleijlevens, Marielle J. van Breemen, Marjan van Meurs, Leonie A. Boven, Jon D. Laman, Mary Teresa Moran, Timothy M. Cox, Johannes M. F. G. Aerts

Blood (2004) 103 (1): 33–39.

https://doi.org/10.1182/blood-2003-05-1612

Published: 2004

Journal Articles

Pathologic gene expression in Gaucher disease: up-regulation of cysteine proteinases including osteoclastic cathepsin K

Mary Teresa Moran, J. Paul Schofield, Alison R. Hayman, Guo-Ping Shi, Elisabeth Young, Timothy M. Cox

Blood (2000) 96 (5): 1969–1978.

https://doi.org/10.1182/blood.V96.5.1969

Published: 2000

NARROW

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

Eliglustat maintains long-term clinical stability in patients with Gaucher disease type 1 stabilized on enzyme therapy

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

Encore - a Randomized, Controlled, Open-Label Non-Inferiority Study Comparing Eliglustat to Imiglucerase in Gaucher Disease Type 1 Patients Stabilized on Enzyme Replacement Therapy: 24-Month Results

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

Gaucher Disease Psychosine Mis-Traffics Endocytosed Cargo to a Non-Functional, Pre-Lysosomal Compartment.

Spectrum of hemojuvelin gene mutations in 1q-linked juvenile hemochromatosis

Screening hepcidin for mutations in juvenile hemochromatosis: identification of a new mutation (C70R)

Marked elevation of the chemokine CCL18/PARC in Gaucher disease: a novel surrogate marker for assessing therapeutic intervention

Pathologic gene expression in Gaucher disease: up-regulation of cysteine proteinases including osteoclastic cathepsin K

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American Society of Hematology

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American Society of Hematology

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