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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

Restore the Secretion and Activity of Factor VIII Mutants By Proteostasis Regulator

Wei Wei, MD PhD, Zhigang Liu, PhD, Bin Zhang, PhD

Blood (2019) 134 (Supplement_1): 1104.

https://doi.org/10.1182/blood-2019-129156

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

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

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

Early Diagnosis of an Infant with Gaucher's Disease Type 3. Case Report

Adriana C. Bello, MD, Rossana Cortez

Blood (2018) 132 (Supplement 1): 4950.

https://doi.org/10.1182/blood-2018-99-110263

Published: 2018

Journal Articles

Identification of GBA Gene Mutations in 19 Pakistani Unrelated Patients of Gaucher Disease

Arshi Naz, PhD, Qurat Abedin, M.Sc, Shariq Ahmed, MPhil, Saima Siddiqui, FCPS, Tahir Shamsi

Blood (2018) 132 (Supplement 1): 4952.

https://doi.org/10.1182/blood-2018-99-120385

Published: 2018

Journal Articles

Flow Cytometry Assay to Monitor Effectiveness of Gene Therapy Correction in Peripheral Blood Mononuclear Cells of Gaucher Disease Type I Patients

Cecilia Barese, MD PhD, Michael Waring, Richard Pfeifer, PhD DABT, Chris Mason, MD PhD

Blood (2018) 132 (Supplement 1): 5791.

https://doi.org/10.1182/blood-2018-99-119766

Published: 2018

Journal Articles

Altering Glycosphingolipid Composition to Improve Multiple Myeloma Bone Complication

Houfu Leng, MSc, DPhil, MBBS, BSc, Adel Ersek, PhD, Emma Morris, DPhil, Beatriz Gamez Molina, PhD, Claire M. Edwards, PhD, Nicole Horwood, PhD

Blood (2018) 132 (Supplement 1): 1942.

https://doi.org/10.1182/blood-2018-99-112237

Published: 2018

Journal Articles

In Vitro Erythropoietic Response to Enzyme Replacement Therapy in Gaucher Type 1 Patients

Paola Delbini, PhD, Irene Motta, Elena Cassinerio, Lorena Duca, Elena Trombetta, Margherita Migone De Amicis, Maria Domenica Cappellini, MD

Blood (2017) 130 (Supplement 1): 3476.

https://doi.org/10.1182/blood.V130.Suppl_1.3476.3476

Published: 2017

Journal Articles

Preliminary Results of Gau-PED Study: Prevalence of Gaucher Disease in Paediatric Patients Selected By an Appropriate Diagnostic Algorithm

William Morello, MD, Mirella Filocamo, Marina Stroppiano, Maja Di Rocco, MD, Nicola Tovaglieri, MD, Giovanna Russo, MD, Francesca Furlan, MD, Roberta Burnelli, MD, Andrea Pession, MD

Blood (2017) 130 (Supplement 1): 1187.

https://doi.org/10.1182/blood.V130.Suppl_1.1187.1187

Published: 2017

Journal Articles

Health-Related Quality of Life of Twenty-Seven Patients with Gaucher Disease Type I in China

Hongmin Li, PhD, Yuan Li, Zhangbiao Long, MD, Yali Du, Miao Chen, Junling Zhuang, Zhengqing Qiu, Zhao Wang, MD PhD, Bing Han

Blood (2017) 130 (Supplement 1): 4812.

https://doi.org/10.1182/blood.V130.Suppl_1.4812.4812

Published: 2017

Journal Articles

A Novel Homozygous CTC1 Germline Mutation Associated with Bone Marrow Failure

Ulla Wartiovaara-Kautto, MD PhD, Elina A. M. Hirvonen, MSc, Kaisa Kettunen, PhD, Anna Nordberg, PhD, Janna Saarela, MD PhD, Kimmo Porkka, MD PhD, Tero Kivelä, MD PhD, Tarja Linnankivi, MD PhD, Outi Kilpivaara, PhD

Blood (2016) 128 (22): 1508.

https://doi.org/10.1182/blood.V128.22.1508.1508

Published: 2016

Journal Articles

Novel Management of Immune Thrombocytopenia in Gaucher Disease Patients

Hanna Rosenbaum, MD

Blood (2014) 124 (21): 5006.

https://doi.org/10.1182/blood.V124.21.5006.5006

Published: 2014

Journal Articles

Assessment of Hematologic and Visceral Parameters in Patients with Type 1 Gaucher Disease in Spain: An Epidemiological Study Using the Therapeutic Goals MAP (Monitor, Action and Progress) Tool ®

Pilar Giraldo, Jordi Perez, Ramiro Núñez, Rafael Fernandez de la Puebla, Elisa Luño, Salvador Saura, Juan Carlos Bureo, Javier de la Serna, Sylvia Plaza, Víctor Navas

Blood (2014) 124 (21): 4963.

https://doi.org/10.1182/blood.V124.21.4963.4963

Published: 2014

NARROW

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

Restore the Secretion and Activity of Factor VIII Mutants By Proteostasis Regulator

Liver-Directed AAV Gene Therapy for Gaucher Disease

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

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

Early Diagnosis of an Infant with Gaucher's Disease Type 3. Case Report

Identification of GBA Gene Mutations in 19 Pakistani Unrelated Patients of Gaucher Disease

Flow Cytometry Assay to Monitor Effectiveness of Gene Therapy Correction in Peripheral Blood Mononuclear Cells of Gaucher Disease Type I Patients

Altering Glycosphingolipid Composition to Improve Multiple Myeloma Bone Complication

In Vitro Erythropoietic Response to Enzyme Replacement Therapy in Gaucher Type 1 Patients

Preliminary Results of Gau-PED Study: Prevalence of Gaucher Disease in Paediatric Patients Selected By an Appropriate Diagnostic Algorithm

Health-Related Quality of Life of Twenty-Seven Patients with Gaucher Disease Type I in China

A Novel Homozygous CTC1 Germline Mutation Associated with Bone Marrow Failure

Novel Management of Immune Thrombocytopenia in Gaucher Disease Patients

Assessment of Hematologic and Visceral Parameters in Patients with Type 1 Gaucher Disease in Spain: An Epidemiological Study Using the Therapeutic Goals MAP (Monitor, Action and Progress) Tool ®

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American Society of Hematology

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ASH Publications

American Society of Hematology

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