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Journal Articles

High-Risk Population Screening for Gaucher Disease: Final Results and Key Learnings from an Italian Multicenter Observational Study

Irene Motta, MD, Christian Benedetto, Marina Stroppiano, Elena Cassinerio, MD, Dario Consonni, MD, Lorenza Borin, MD, Paola Ranalli, MD, Wilma Barcellini, Maria Domenica Cappellini, MD

Blood (2020) 136 (Supplement 1): 17–18.

https://doi.org/10.1182/blood-2020-141527

Published: 2020

Journal Articles

Diagnostic Work-up of Pulmonary Hypertension in Non Transfusion Dependent Thalassemia Patients: Pathophysiologichal Mechanisms and Clinical Implications

Marta Mancarella, MD, Irene Motta, MD, Irene Rota, MD, Margherita Migone De Amicis, MD, Alessia Marcon, MD, Marta Ferraresi, MD, Marianna Giuditta, MD, Giovanna Graziadei, MD, Maria Domenica Cappellini, MD, Marco Vicenzi, MD

Blood (2020) 136 (Supplement 1): 21–22.

https://doi.org/10.1182/blood-2020-140819

Published: 2020

Journal Articles

COVID 19 and Hemoglobinopathies: Update of the Italian Experience

Irene Motta, MD, Valeria Maria Pinto, MD, Filomena Longo, MD, Federico Bonetti, MD, Elisa De Michele, MD, Alberto Piperno, MD, Lorella Pitrolo, MD, Micol Quaresima, MD, Michela Ribersani, MD, Margherita Migone De Amicis, MD, Manuela Balocco, MD, Barbara Gianesin, PhD, Giovanna Graziadei, MD, Maria Domenica Cappellini, MD, Lucia De Franceschi, MD, Antonio Piga, MD, Gian Luca Forni, MD

Blood (2020) 136 (Supplement 1): 17–18.

https://doi.org/10.1182/blood-2020-141610

Published: 2020

Journal Articles

Inhibition of Fibroblast Growth Factor-23 (FGF-23) As a Novel Strategy to Target Bone and Hematopoietic Stem Cell Niche Defects in Beta-Thalassemia

Annamaria Aprile, PhD, Laura Raggi, Mariangela Storto, Isabella Villa, PhD, Sarah Marktel, MD, Irene Motta, MD, Maria Domenica Cappellini, Alessandro Rubinacci, MD, Giuliana Ferrari, PhD

Blood (2020) 136 (Supplement 1): 2.

https://doi.org/10.1182/blood-2020-137461

Published: 2020

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

Iron Chelation and Ferritin below 500 Mcg/L in Transfusion Dependent Thalassemia: Beyond the Limits of Clinical Trials

Natalia Scaramellini, MD, Carola Arighi, MD, Alessia Marcon, MD, Dario Consonni, MD, Elena Cassinerio, MD, Giovanna Graziadei, MD, Maria Domenica Cappellini, MD, Irene Motta, MD

Blood (2019) 134 (Supplement_1): 3542.

https://doi.org/10.1182/blood-2019-130237

Published: 2019

Journal Articles

2'-O-Methoxyethyl Splice-Switching Oligos to Reverse Splicing from IVS2-745 β-Thalassemia Patient Cells: A Foundation for Potential Therapies

Blood (2019) 134 (Supplement_1): 2244.

https://doi.org/10.1182/blood-2019-127017

Published: 2019

Journal Articles

Activation of ULK1 Kinase Mediates Clearance of Free Alpha-Globin in Human Beta-Thalassemic Erythroblasts

Christophe Lechauve, PhD, Julia Keith, Eugene Khandros, MD PhD, Stephanie Fowler, Kalin Mayberry, Abdullah Freiwan, Christopher Thom, MD PhD, Jingjing Zhang, Heather Tillman, DVM, PhD, Mondira Kundu, MD PhD, Irene Motta, MD, Maria Domenica Cappellini, MD, Mitchell J. Weiss, MD PhD

Blood (2018) 132 (Supplement 1): 411.

https://doi.org/10.1182/blood-2018-99-113901

Published: 2018

Journal Articles

Adult Hemoglobin Production, Chain Rebalance, and Splice Correction in IVS2-745 Beta-Thalassemia Patient Cells Using 2'-O-Methoxyethyl Splice-Switching Oligos

Alisa Cheung Dong, Valentina Ghiaccio, Irene Motta, MD, Shuling Guo, PhD, Raechel Peralta, MS, Coralea Stephanou, PhD, Paola Delbini, PhD, Connie Chen, Soteroula Christou, Marina Kleanthous, PhD, Maria Domenica Cappellini, MD, Osheiza Abdulmalik, DVM, MS, Laura Breda, PhD, Stefano Rivella, PhD

Blood (2016) 128 (22): 1014.

https://doi.org/10.1182/blood.V128.22.1014.1014

Published: 2016

Journal Articles

Adrenal Insufficiency: An Emerging Challenge in Thalassemia?

Marta Mancarella, MD, Diletta Maira, MD, Valeria Ferla, Marco Capecchi, MD, Irene Motta, MD, Elena Cassinerio, MD, Alessia Marcon, MD, Marina Baldini, MD, Maria Domenica Cappellini, MD

Blood (2016) 128 (22): 4830.

https://doi.org/10.1182/blood.V128.22.4830.4830

Published: 2016

Journal Articles

Comparing Strategies to Reactivate Fetal Globin Expression for the Treatment of Beta-Globinopathies

Laura Breda, PhD, Jeremy W. Rupon, MD PhD, Irene Motta, MD, Wulan Deng, Gerd A. Blobel, MD PhD, Stefano Rivella, PhD

Blood (2014) 124 (21): 333.

https://doi.org/10.1182/blood.V124.21.333.333

Published: 2014

Journal Articles

A Multicenter Observational Study For Early Diagnosis Of Gaucher Disease In Patients With Splenomegaly and/Or Thrombocytopenia

Irene Motta, MD, Mirella Filocamo, BSc, PhD, Marina Stroppiano, BSc, PhD, Erika Poggiali, MD, Alfredo Dragani, MD, Maria Domenica, MD

Blood (2013) 122 (21): 4712.

https://doi.org/10.1182/blood.V122.21.4712.4712

Published: 2013

Journal Articles

Iron Metabolism In Macrophages In Non Transfusion Dependent Thalassemia

Valentina Vaja, PhD, Elena Paltrinieri, MSc, Irene Motta, MD, Erika Poggiali, MD, Alessia Marcon, MD, Giovanna Graziadei, MD, Marina Zarantonello, Maria Domenica Cappellini, MD

Blood (2013) 122 (21): 4669.

https://doi.org/10.1182/blood.V122.21.4669.4669

Published: 2013

NARROW

High-Risk Population Screening for Gaucher Disease: Final Results and Key Learnings from an Italian Multicenter Observational Study

Diagnostic Work-up of Pulmonary Hypertension in Non Transfusion Dependent Thalassemia Patients: Pathophysiologichal Mechanisms and Clinical Implications

COVID 19 and Hemoglobinopathies: Update of the Italian Experience

Inhibition of Fibroblast Growth Factor-23 (FGF-23) As a Novel Strategy to Target Bone and Hematopoietic Stem Cell Niche Defects in Beta-Thalassemia

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

Iron Chelation and Ferritin below 500 Mcg/L in Transfusion Dependent Thalassemia: Beyond the Limits of Clinical Trials

2'-O-Methoxyethyl Splice-Switching Oligos to Reverse Splicing from IVS2-745 β-Thalassemia Patient Cells: A Foundation for Potential Therapies

Activation of ULK1 Kinase Mediates Clearance of Free Alpha-Globin in Human Beta-Thalassemic Erythroblasts

Adult Hemoglobin Production, Chain Rebalance, and Splice Correction in IVS2-745 Beta-Thalassemia Patient Cells Using 2'-O-Methoxyethyl Splice-Switching Oligos

Adrenal Insufficiency: An Emerging Challenge in Thalassemia?

Comparing Strategies to Reactivate Fetal Globin Expression for the Treatment of Beta-Globinopathies

A Multicenter Observational Study For Early Diagnosis Of Gaucher Disease In Patients With Splenomegaly and/Or Thrombocytopenia

Iron Metabolism In Macrophages In Non Transfusion Dependent Thalassemia

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American Society of Hematology

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American Society of Hematology

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