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Journal Articles

Management Algorithms for Gaucher Disease Type 1 in Saudi Arabia: A Consensus Result from a National Meeting

Ayman Alhejazi, MD, Abdul Kareem Almomen, MD, Ahmad M Tarawah, Ahmed M AlSuliman, Hussain H. Al Saeed, MBBS, MD, Mahsen AlSaleh, MD, Ohoud F Kashari, Marwan Elbagoury, MSc, Omar Hussein

Blood (2019) 134 (Supplement_1): 4864.

https://doi.org/10.1182/blood-2019-122822

Published: 2019

Journal Articles

Multidisciplinary Study Based on Clinical, Electrophysiological and Psycological Evaluations Combined with Advanced Neuroimaging in Gaucher Disease Patients

Fiorina Giona, MD, Carlo Di Bonaventura, MD, Patrizia Mancini, MD, Gianmarco Tessari, Turchetta Rosaria, MD, Emanuele Cerulli Irelli, MD, Stefano Ferracuti, Teresa Di Giallonardo, MD, Rocco Plateroti, MD, Francesca Atturo, MD, Luisa Cardarelli, MD, Giovanna Palumbo, MD, Marcella Nebbioso, MD, Tiziana Di Pippo, Francesca Caramia, MD

Blood (2019) 134 (Supplement_1): 2185.

https://doi.org/10.1182/blood-2019-128143

Published: 2019

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

Bone Marrow Plasma Cell Burden in Gaucher Disease Correlates with Local Macrophage Infiltration and May be Altered By Disease Treatment

Monika Klimkowska, MD PhD, Maciej J. Machaczka, MD PhD

Blood (2019) 134 (Supplement_1): 3593.

https://doi.org/10.1182/blood-2019-131343

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

Computational Analysis Model Applied to Spanish Gaucher Registry Data

Marcio M Andrade-Campos, MD PhD, Laura Lopez de Frutos, PhD, Jorge J Cebolla, MSc, Blanca Medrano-Engay, PhD, Mercedes Roca-Espiau, PhD, Beatriz Gomez-Barrera, David Iniguez, Pilar Giraldo, PhD

Blood (2019) 134 (Supplement_1): 2323.

https://doi.org/10.1182/blood-2019-122347

Published: 2019

Journal Articles

Not All Patients with Mild Phenotype of Gaucher Disease (GD) Need Disease Specific Treatment As Reflected By Patients Reported Outcomes Measures

Shoshana Revel-Vilk, MD, Tama Dinur, PhD, Majdolen Istaiti, Dafna Frydman, DMV, Michal Becker-Cohen, MSc, Ari Zimran, MD

Blood (2019) 134 (Supplement_1): 4712.

https://doi.org/10.1182/blood-2019-123000

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

Bone Marrow Involvement in Patients with Type 1 Gaucher Disease Receiving Low-Dose Long Term Enzyme Replacement Therapy (median 18 years)

Shoshana Revel-Vilk, MD, Jeffrey Szer, MBBS, Michal Becker Cohen, Ari Zimran, MD

Blood (2019) 134 (Supplement_1): 4862.

https://doi.org/10.1182/blood-2019-131013

Published: 2019

Journal Articles

Polyunsaturated Fatty Acid (PUFA) Signaling Induces Ferroptosis-Mediated Cell-Death in Multiple Myeloma

Cristina Panaroni, PhD, Keertik Fulzele, PhD, Rosemary Soucy, Cherrie Huang, Kenta Mukaihara, MD PhD, Shrikanta Chattopadhyay, MBBS, Noopur S. Raje, MD

Blood (2019) 134 (Supplement_1): 3108.

https://doi.org/10.1182/blood-2019-131906

Published: 2019

Journal Articles

Liver-Directed AAV Gene Therapy for Gaucher Disease

Carlos J Miranda, PhD, Miriam Canavese, PhD, Elisa Chisari, MS, Jalpa Pandya, MPhil, Clement Cocita, PhD, Maria Portillo, MRes, Jenny McIntosh, PhD, Azadeh Kia, PhD, Jonathan H. Foley, PhD, Allison Dane, PhD, Jey M Jeyakumar, PhD, Rose Sheridan, PhD, Romuald Corbau, Amit C. Nathwani, MD PhD

Blood (2019) 134 (Supplement_1): 3354.

https://doi.org/10.1182/blood-2019-124280

Published: 2019

Journal Articles

Plasma cell myeloma in a patient with Gaucher disease

Jeanette M. Ramos, Aubrey J. Hough

Blood (2019) 133 (1): 100.

https://doi.org/10.1182/blood-2018-09-875435

Published: 2019

Journal Articles

Open-Label, Expanded Access Study of Taliglucerase Alfa in Patients with Gaucher Disease Requiring Enzyme Replacement Therapy

David J. Kuter, MD DPhil, Michael Wajnrajch, MD MPA, Betina Hernandez, MD, Rong Wang, MPH, Raul Chertkoff, MD MBA, Ari Zimran, MD

Blood (2018) 132 (Supplement 1): 3755.

https://doi.org/10.1182/blood-2018-99-112660

Published: 2018

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

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

MDS/Secondary AML Associated with Type I Gaucher Disease

Atsushi Okamoto, MD, Kenta Yamamoto, Go Eguchi, Yoshitaka Kanai, Terufumi Yamaguchi, Yasuhiro Maeda, MD PhD

Blood (2018) 132 (Supplement 1): 5193.

https://doi.org/10.1182/blood-2018-99-116058

Published: 2018

Journal Articles

Arachidonic Acid Induces Ferroptosis-Mediated Cell-Death in Multiple Myeloma

Cristina Panaroni, PhD, Keertik Fulzele, PhD, Rosemary Soucy, Ka Tat Siu, PhD, Kenta Mukaihara, MD PhD, Cherrie Huang, Shrikanta Chattopadhyay, MBBS, Noopur Raje, MD

Blood (2018) 132 (Supplement 1): 4498.

https://doi.org/10.1182/blood-2018-99-118482

Published: 2018

Journal Articles

Twenty-Five Years Diagnosing Gaucher’s Disease in Spain, What We Have Learned?

Marcio M Andrade-Campos, PhD, Jose L Capablo, PhD, Jesus J Fraile, PhD, Mercedes Roca-Espiau, PhD, Pilar Irun, PhD, Jorge J Cebolla, MSc, Laura Lopez de Frutos, PhD, Izaskun Arenaz, MSci, Jesus Villarrubia, PhD, Abelardo Barez, PhD, Horacio Cano, MD, Teresa Molero, PhD, Lucia Villalon, PhD, Pilar Giraldo, PhD

Blood (2018) 132 (Supplement 1): 1110.

https://doi.org/10.1182/blood-2018-99-115474

Published: 2018

Journal Articles

Prospective National-Base Multicenter Study to Standardize the Follow-up of Type 1Gaucher Disease Patients Treated with Eliglustat Under Standard of Care Practice. Trazelga Project

Marcio M Andrade-Campos, PhD, Jorge J Cebolla, MSc, Laura Lopez de Frutos, PhD, Pilar Irun, PhD, Soledad Noya, MD, Santiago Nieto, MD, Lucia Villalon, PhD, Ana I Arribas, MD, Abelardo Barez, PhD, Javier Garcia-Frade, PD, Maria Lourdes Hermosin, MD, Jesús-María Hernández-Rivas, MD PhD, Daiana Ibarretxe, MD, Maria L Lozano, MD PhD, Teresa Molero, PhD, Maria Angeles Perez-Saenz, MDPhD, Jesus Villarrubia, PhD, Pilar Giraldo, PhD

Blood (2018) 132 (Supplement 1): 4942.

https://doi.org/10.1182/blood-2018-99-115534

Published: 2018

Journal Articles

The Challenge of Unexplained Splenomegalies: Preliminary Data of the French Prospective Multi-Center Splenomegaly Study (SMS)

Guillaume Denis, MD, Laurence Sanhes, MD, Jean-Marc Ziza, MD PhD, Frederic Bauduer, MD PhD, Marc G. Berger, MD PhD, Regis T. Costello, MD PhD, Kawkab Mehdi, Martin Michaud, MD, Geoffrey Urbanski, MD, Christian Rose, MD PhD

Blood (2018) 132 (Supplement 1): 4815.

https://doi.org/10.1182/blood-2018-99-113123

Published: 2018

NARROW

Management Algorithms for Gaucher Disease Type 1 in Saudi Arabia: A Consensus Result from a National Meeting

Multidisciplinary Study Based on Clinical, Electrophysiological and Psycological Evaluations Combined with Advanced Neuroimaging in Gaucher Disease Patients

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

Bone Marrow Plasma Cell Burden in Gaucher Disease Correlates with Local Macrophage Infiltration and May be Altered By Disease Treatment

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

Computational Analysis Model Applied to Spanish Gaucher Registry Data

Not All Patients with Mild Phenotype of Gaucher Disease (GD) Need Disease Specific Treatment As Reflected By Patients Reported Outcomes Measures

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

Bone Marrow Involvement in Patients with Type 1 Gaucher Disease Receiving Low-Dose Long Term Enzyme Replacement Therapy (median 18 years)

Polyunsaturated Fatty Acid (PUFA) Signaling Induces Ferroptosis-Mediated Cell-Death in Multiple Myeloma

Liver-Directed AAV Gene Therapy for Gaucher Disease

Plasma cell myeloma in a patient with Gaucher disease

Open-Label, Expanded Access Study of Taliglucerase Alfa in Patients with Gaucher Disease Requiring Enzyme Replacement Therapy

Long-Term Effects of Oral Eliglustat on Skeletal Manifestations of Gaucher Disease Type 1: Results from Four 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

MDS/Secondary AML Associated with Type I Gaucher Disease

Arachidonic Acid Induces Ferroptosis-Mediated Cell-Death in Multiple Myeloma

Twenty-Five Years Diagnosing Gaucher’s Disease in Spain, What We Have Learned?

Prospective National-Base Multicenter Study to Standardize the Follow-up of Type 1Gaucher Disease Patients Treated with Eliglustat Under Standard of Care Practice. Trazelga Project

The Challenge of Unexplained Splenomegalies: Preliminary Data of the French Prospective Multi-Center Splenomegaly Study (SMS)

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American Society of Hematology

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ASH Publications

American Society of Hematology

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