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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 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

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

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

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

Chronic Myeloid Leukaemia Patients with Stable Molecular Responses (at least MR3) May Safely Decrease the Dose of Their Tyrosine Kinase Inhibitor: Data from the British Destiny Study

Richard E. Clark, MD, Fotios Polydoros, MSc, Jane F. Apperley, MD, Christopher Pocock, Graeme Smith, MD, Jenny L Byrne, FRCP FRCPath PhD, Stephen G. O'Brien, BSc (Hons) PhD MBChB FRCP MRCPath, Tony Coffey, Letizia Foroni, MD PhD, Mhairi Copland, BSc (Hons) MB ChB PhDFRCP FRCPath

Blood (2016) 128 (22): 938.

https://doi.org/10.1182/blood.V128.22.938.938

Published: 2016

Journal Articles

Neonatal Bone Marrow Transplantation Prevents Bone Pathology in a Mouse Model of Mucopolysaccharidosis Type I

Alice Pievani, PhD, Isabella Maria Rebecca Azario, Laura Antolini, Tsutomu Shimada, Pravin Patel, Cristina Remoli, Benedetta Rambaldi, Maria Grazia Valsecchi, Mara Riminucci, Andrea Biondi, Shunji Tomatsu, Marta Serafini

Blood (2014) 124 (21): 649.

https://doi.org/10.1182/blood.V124.21.649.649

Published: 2014

Journal Articles

Survival Outcomes of Double and Triple-Only Combination Anti-Myeloma Chemotherapeutic Regimens on 26 Multiple Myeloma Patients Seen in a Sub-Saharan African Country

Ogbonna Collins Nwabuko, MBBS, FMCPath, Martin Anazodo Nnoli, MBBS, MSc, FMCPath., Elizabeth Eneikido Igbigbi, MBBS, FMCPath, Dorathy Adaunwa Okoh, MBBS, FWACP,FMCPath, Ijezie Innocent Chukwuonye, MBBS, FMCP

Blood (2014) 124 (21): 5762.

https://doi.org/10.1182/blood.V124.21.5762.5762

Published: 2014

Journal Articles

Eliglustat, an Investigational Oral Therapy for Gaucher Disease Type 1: Phase 2 Results After 4 Years of Treatment

M. Judith Peterschmitt, MD, Elena Lukina, PhD, MD, Nora Watman, MD, Marta Dragosky, M.D., Gregory M Pastores, Elsa Avila Arreguin, M.D., Hanna Rosenbaum, MD, Ari Zimran, MD, Elena Sysoeva, Rasha Aguzzi, Leorah H Ross, Ana Cristina Puga, M.D.

Blood (2012) 120 (21): 1038.

https://doi.org/10.1182/blood.V120.21.1038.1038

Published: 2012

Journal Articles

Bone Involvement in Patients with Non-Neuronopathic Gaucher Disease Is Characterized by Increased Osteoclast Function Partially Due to High Circulating C-C Motif Ligand 3,

Veroniki Komninaka, Theodore Marinakis, Dimitrios Christoulas, Konstantina Repa, Dionysia Kolomodi, Maria Mitropoulou, Maria Gkotzamanidou, Athanasios Papatheodorou, Nikolaos Anagnostopoulos, Meletios Athanasios Dimopoulos, MD, Evangelos Terpos

Blood (2011) 118 (21): 3215.

https://doi.org/10.1182/blood.V118.21.3215.3215

Published: 2011

Journal Articles

Improvements in Skeletal Manifestations in Gaucher Disease Type 1 Patients After 3 Years of Treatment with Oral Eliglustat During a Phase 2 Trial,

M. Judith Peterschmitt, MD, Ravi S Kamath, MD, Elena Lukina, PhD, MD, Nora Watman, MD, Marta Dragosky, M.D., Marcelo Iastrebner, M.D., Gregory M Pastores, Elsa Avila Arreguin, M.D., Mici Phillips, M.D., Hanna Rosenbaum, MD, Elena Sysoeva, Rasha Aguzzi, Leorah H Ross, Ana Cristina Puga, M.D., Daniel I Rosenthal, M.D.

Blood (2011) 118 (21): 3216.

https://doi.org/10.1182/blood.V118.21.3216.3216

Published: 2011

Journal Articles

Lukina E, Watman N, Arreguin EA, et al. Improvement in hematological, visceral, and skeletal manifestations of Gaucher disease type 1 with oral eliglustat tartrate (Genz-112638) treatment: 2-year results of a phase 2 study. Blood . 2010;116(20): 4095–4098 .

Blood (2011) 117 (20): 5551.

https://doi.org/10.1182/blood-2011-04-347146

Published: 2011

Journal Articles

Improvement in hematological, visceral, and skeletal manifestations of Gaucher disease type 1 with oral eliglustat tartrate (Genz-112638) treatment: 2-year results of a phase 2 study

Elena Lukina, Nora Watman, Elsa Avila Arreguin, Marta Dragosky, Marcelo Iastrebner, Hanna Rosenbaum, Mici Phillips, Gregory M. Pastores, Ravi S. Kamath, Daniel I. Rosenthal, Mathilde Kaper, Tejdip Singh, Ana Cristina Puga, M. Judith Peterschmitt

Blood (2010) 116 (20): 4095–4098.

https://doi.org/10.1182/blood-2010-06-293902

Published: 2010

Journal Articles

Inherited Failure Syndromes.

Grover C. Bagby, Jr., MD

Blood (2009) 114 (22): SCI-10.

https://doi.org/10.1182/blood.V114.22.SCI-10.SCI-10

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

Blood (2008) 112 (11): 4648.

https://doi.org/10.1182/blood.V112.11.4648.4648

Published: 2008

Journal Articles

The Clinical Course of Untreated Gaucher Disease in 22 Patients Over 10 Years: Hematological and Skeletal Manifestations.

Siavash Piran, MSc, Andrew Roberts, BSc, Mary Anne Patterson, RN, Dominick Amato, MD

Blood (2008) 112 (11): 1264.

https://doi.org/10.1182/blood.V112.11.1264.1264

Published: 2008

Journal Articles

Gaucher Disease: A Systematic Review and Meta-Analysis of Bone Complications and Their Response to Treatment.

Siavash Piran, MSc, Dominick Amato, MD

Blood (2008) 112 (11): 1265.

https://doi.org/10.1182/blood.V112.11.1265.1265

Published: 2008

Journal Articles

Molecular Responses in Newly Diagnosed Chronic Myelocytic Leukemia (CML) Patients Treated with 800 mg Imatinib Daily: An Update from the RIGHT Trial Study Group.

Jorge Cortes, MD, Francis Giles, MD, August J. Salvado, MD, Karen McDougall, Jerald Radich, MD, Maher Albitar, MD, Bayard L. Powell, MD, Stuart Goldberg, MD, Hagop M. Kantarjian, MD

Blood (2006) 108 (11): 2149.

https://doi.org/10.1182/blood.V108.11.2149.2149

Published: 2006

Journal Articles

Artificially Induced Thyroid Suppression in Sickle Cell Disease

Charles W. Seward, M.D., John W. Eaton, Ph.D., Hugh Chaplin, Jr., M.D.

Blood (1972) 40 (6): 905–913.

https://doi.org/10.1182/blood.V40.6.905.905

Published: 1972

NARROW

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

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

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

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

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

Chronic Myeloid Leukaemia Patients with Stable Molecular Responses (at least MR3) May Safely Decrease the Dose of Their Tyrosine Kinase Inhibitor: Data from the British Destiny Study

Neonatal Bone Marrow Transplantation Prevents Bone Pathology in a Mouse Model of Mucopolysaccharidosis Type I

Survival Outcomes of Double and Triple-Only Combination Anti-Myeloma Chemotherapeutic Regimens on 26 Multiple Myeloma Patients Seen in a Sub-Saharan African Country

Eliglustat, an Investigational Oral Therapy for Gaucher Disease Type 1: Phase 2 Results After 4 Years of Treatment

Bone Involvement in Patients with Non-Neuronopathic Gaucher Disease Is Characterized by Increased Osteoclast Function Partially Due to High Circulating C-C Motif Ligand 3,

Improvements in Skeletal Manifestations in Gaucher Disease Type 1 Patients After 3 Years of Treatment with Oral Eliglustat During a Phase 2 Trial,

Lukina E, Watman N, Arreguin EA, et al. Improvement in hematological, visceral, and skeletal manifestations of Gaucher disease type 1 with oral eliglustat tartrate (Genz-112638) treatment: 2-year results of a phase 2 study. Blood . 2010;116(20): 4095–4098 .

Improvement in hematological, visceral, and skeletal manifestations of Gaucher disease type 1 with oral eliglustat tartrate (Genz-112638) treatment: 2-year results of a phase 2 study

Inherited Failure Syndromes.

Diagnostic and Disease Management Algorithms for Gaucher Disease: A Guide for Haematologists

The Clinical Course of Untreated Gaucher Disease in 22 Patients Over 10 Years: Hematological and Skeletal Manifestations.

Gaucher Disease: A Systematic Review and Meta-Analysis of Bone Complications and Their Response to Treatment.

Molecular Responses in Newly Diagnosed Chronic Myelocytic Leukemia (CML) Patients Treated with 800 mg Imatinib Daily: An Update from the RIGHT Trial Study Group.

Artificially Induced Thyroid Suppression in Sickle Cell Disease

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American Society of Hematology

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American Society of Hematology

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