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Journal Articles

The Association of Anemia and Patient Reported Outcomes in the New Era of PNH Treatment

Blood (2024) 144 (Supplement 1): 2321.

https://doi.org/10.1182/blood-2024-204693

Published: 2024

Journal Articles

Factor B Inhibition with Oral Iptacopan Monotherapy Demonstrates Sustained Long-Term Efficacy and Safety in Anti-C5-Treated Patients (pts) with Paroxysmal Nocturnal Hemoglobinuria (PNH) and Persistent Anemia: Final 48-Week Results from the Multicenter, Phase III APPLY-PNH Trial

Blood (2023) 142 (Supplement 1): 571.

https://doi.org/10.1182/blood-2023-180780

Published: 2023

Journal Articles

Ravulizumab Provides Durable Control of Intravascular Hemolysis and Improves Survival in Patients with Paroxysmal Nocturnal Hemoglobinuria: Long-Term Follow-up of Study 301 and Comparisons with Patients of the International PNH Registry

Austin Kulasekararaj, Hubert Schrezenmeier, Kensuke Usuki, Aleksander Kulagin, Sandra Fatima Gualandro, Rosario Notaro, Masayo Ogawa, Ji Yu, Ami Patel, Yogesh Patel, Wolfgang Füreder, Regis Peffault De Latour, Jong-Wook Lee

Blood (2023) 142 (Supplement 1): 2714.

https://doi.org/10.1182/blood-2023-189150

Published: 2023

Journal Articles

Vemircopan (ALXN2050) Monotherapy in Paroxysmal Nocturnal Hemoglobinuria: Interim Data from a Phase 2 Open-Label Proof-of-Concept Study

Peter J. Browett, Austin Kulasekararaj, Rosario Notaro, Masayo Ogawa, Antonio Risitano, Ji Yu, Jong Wook Lee

Blood (2022) 140 (Supplement 1): 717–719.

https://doi.org/10.1182/blood-2022-169301

Published: 2022

Journal Articles

Phase 2 study of danicopan in patients with paroxysmal nocturnal hemoglobinuria with an inadequate response to eculizumab

Austin G. Kulasekararaj, Antonio M. Risitano, Jaroslaw P. Maciejewski, Rosario Notaro, Peter Browett, Jong Wook Lee, Mingjun Huang, Michael Geffner, Robert A. Brodsky

Blood (2021) 138 (20): 1928–1938.

https://doi.org/10.1182/blood.2021011388

Published: 2021

Includes: Supplemental data

Journal Articles

Complement-mediated oxidative damage of red cells impairs response to eculizumab in a G6PD-deficient patient with PNH

Michela Sica, Annamaria Pellecchia, Maria De Angioletti, Giovanni Caocci, Caterina Nannelli, Giorgio La Nasa, Lucio Luzzatto, Rosario Notaro

Blood (2020) 136 (26): 3082–3085.

https://doi.org/10.1182/blood.2020007780

Published: 2020

Includes: Supplemental data

Journal Articles

COVID-19 Infection in Patients with Paroxysmal Nocturnal Hemoglobinuria in Italy

Wilma Barcellini, Bruno Fattizzo, MD, Luisa Quattrocchi, Juri Alessandro Giannotta, MD, Semra Aydin, MD PhD, Federica Barone, MD, Cecilia Carbone, MD, Eloise Beggiato, Paola Bianchi, BSc, PhD, Eros Di Bona, MD, Rosario Notaro, MD, Anna Paola Iori

Blood (2020) 136 (Supplement 1): 36–37.

https://doi.org/10.1182/blood-2020-138721

Published: 2020

Journal Articles

Efficacy and Safety of Concomitant Use of Ravulizumab and IST in Patients with Paroxysmal Nocturnal Hemoglobinuria up to 52 Weeks

Hubert Schrezenmeier, Jong Wook Lee, MD, PhD, Anita Hill, Vadim V Ptushkin, Viviani Pessoa, Rosario Notaro, Jimmy Wang, Masayo Ogawa, MD FAAP, Shinichiro Okamoto, MD PhD, Lily LL Wong, MD FRCPath, Regis Peffault De Latour, MD PhD, Austin Kulasekararaj, MDMBBS,MRCP,FRCPath

Blood (2020) 136 (Supplement 1): 37–38.

https://doi.org/10.1182/blood-2020-142453

Published: 2020

Journal Articles

Glucose-6-phosphate dehydrogenase deficiency

Lucio Luzzatto, Mwashungi Ally, Rosario Notaro

Blood (2020) 136 (11): 1225–1240.

https://doi.org/10.1182/blood.2019000944

Published: 2020

Includes: Supplemental data

Journal Articles

A Phase 2 Open-Label Study of Danicopan (ACH-0144471) in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have an Inadequate Response to Eculizumab Monotherapy

Austin Kulasekararaj, MBBS MDMRCP FRCPath, Antonio M. Risitano, MD PhD, Jaroslaw P. Maciejewski, MD PhD, Rosario Notaro, MD, Peter Browett, BMedSci, MBChB, FRACP, FRCPA, Jong-Wook Lee, MD, Mingjun Huang, PhD, Michael Geffner, MD, Robert A. Brodsky, MD

Blood (2019) 134 (Supplement_1): 3514.

https://doi.org/10.1182/blood-2019-124748

Published: 2019

Journal Articles

Hematological Response to Eculizumab in Paroxysmal Nocturnal Hemoglobinuria: Application of a Novel Classification to Identify Unmet Clinical Needs and Future Clinical Goals

Pierre-Edouard Debureaux, MD, Fabiana Cacace, Bruno G.P. Silva, MD, Federica Barone, MD, Rodrigo T Calado, MD, Flore Sicre de Fontbrune, Juliette Soret-Dulphy, MD, Patrizia Ricci, Michela Sica, PhD, Rosario Notaro, MD, Phillip Scheinberg, MD, Austin Kulasekararaj, MDMBBS,MRCP,FRCPath, Antonio M. Risitano, MD PhD, Régis Peffault de Latour, MD PhD, Camilla Frieri, MD

Blood (2019) 134 (Supplement_1): 3517.

https://doi.org/10.1182/blood-2019-125917

Published: 2019

Journal Articles

Mechanistic Evaluation of Efficacy Using Biomarkers of the Oral, Small Molecule Factor D Inhibitor, Danicopan (ACH-4471), in Untreated Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)

Antonio M. Risitano, MD PhD, Jong Wook Lee, MD PhD, Rosario Notaro, MD, Robert A. Brodsky, MD, Austin Kulasekararaj, MBBS MDMRCP FRCPath, Jaroslaw P. Maciejewski, MD PhD, Mingjun Huang, PhD, Michael Geffner, MD, Peter Browett, BMedSci, MBChB, FRACP, FRCPA

Blood (2019) 134 (Supplement_1): 2226.

https://doi.org/10.1182/blood-2019-124837

Published: 2019

Journal Articles

Two Distinct but Functionally Related Autoreactive T Cell Populations in Paroxysmal Nocturnal Hemoglobinuria and Idiopathic Aplastic Anemia: Glycosylphosphatidylinositol-Specific and IFNγ-Producing T Cells

Lucia Gargiulo, PhD, Michela Sica, PhD, Caterina Nannelli, BSc, Lucio Luzzatto, MD, Rosario Notaro, MD

Blood (2017) 130 (Supplement 1): 1166.

https://doi.org/10.1182/blood.V130.Suppl_1.1166.1166

Published: 2017

Journal Articles

Glycosylphosphatidylinositol-specific T cells, IFN-γ-producing T cells, and pathogenesis of idiopathic aplastic anemia

Lucia Gargiulo, Yoshitaka Zaimoku, Barbara Scappini, Hiroyuki Maruyama, Rie Ohumi, Lucio Luzzatto, Shinji Nakao, Rosario Notaro

Blood (2017) 129 (3): 388–392.

https://doi.org/10.1182/blood-2016-09-740845

Published: 2017

Journal Articles

CD59 Deficiency Is Critical for C3 Binding on Red Blood Cells of Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) during Anti-C5 Treatment (eculizumab)

MIchela Sica, PhD, Tommaso Rondelli, Patrizia Ricci, PhD, Maria De Angioletti, PhD, Antonio M Risitano, MD PhD, Rosario Notaro, MD

Blood (2016) 128 (22): 401.

https://doi.org/10.1182/blood.V128.22.401.401

Published: 2016

Journal Articles

Auto-Reactive Glycosyl-Phosphatidyl-Inositol (GPI) Specific T Cells Are Present in Most Patients with Idiopathic Aplastic Anemia (IAA)

Lucia Gargiulo, PhD, Yoshitaka Zaimoku, MD, Barbara Scappini, MD, Hiroyuki Maruyama, MD, Rie Ohumi, Lucio Luzzatto, MD, Shinji Nakao, MD PhD, Rosario Notaro, MD

Blood (2015) 126 (23): 2409.

https://doi.org/10.1182/blood.V126.23.2409.2409

Published: 2015

Journal Articles

Complement Activation in Paroxysmal Nocturnal Hemoglobinuria (PNH) Causes Oxidative Damage Which May Affect Response to Eculizumab

Michela Sica, PhD, Annamaria Pellecchia, PhD, Margherita Berardi, BS, Lucia Gargiulo, PhD, Tolulase A Olutogun, MD, Giovanni Caocci, MD, Giorgio La Nasa, MD, Maria De Angioletti, PhD, Lucio Luzzatto, MD FRCP, FRCPath, Rosario Notaro, MD

Blood (2014) 124 (21): 4397.

https://doi.org/10.1182/blood.V124.21.4397.4397

Published: 2014

Journal Articles

Peptide inhibitors of C3 activation as a novel strategy of complement inhibition for the treatment of paroxysmal nocturnal hemoglobinuria

Antonio M. Risitano, Daniel Ricklin, Yijun Huang, Edimara S. Reis, Hui Chen, Patrizia Ricci, Zhuoer Lin, Caterina Pascariello, Maddalena Raia, Michela Sica, Luigi Del Vecchio, Fabrizio Pane, Florea Lupu, Rosario Notaro, Ranillo R. G. Resuello, Robert A. DeAngelis, John D. Lambris

Blood (2014) 123 (13): 2094–2101.

https://doi.org/10.1182/blood-2013-11-536573

Published: 2014

Includes: Supplemental data

Journal Articles

Glycosylphosphatidylinositol-specific, CD1d-restricted T cells in paroxysmal nocturnal hemoglobinuria

Lucia Gargiulo, Maria Papaioannou, Michela Sica, Giulia Talini, Aristeidis Chaidos, Barbara Richichi, Andrei V. Nikolaev, Cristina Nativi, Mark Layton, Josu de la Fuente, Irene Roberts, Lucio Luzzatto, Rosario Notaro, Anastasios Karadimitris

Blood (2013) 121 (14): 2753–2761.

https://doi.org/10.1182/blood-2012-11-469353

Published: 2013

Includes: Supplemental data

Journal Articles

Autoreactive T Cells From Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Specifically Recognize Glycosyl- Phosphatidyl-Inositol (GPI)

Lucia Gargiulo, PhD, Michela Sica, PhD, Maria Papaioannou, MD, PhD, Giulia Talini, PhD, Aristeidis Chaidos, MD, PhD, Barbara Richichi, PhD, Andrei V Nikolaev, PhD, Cristina Nativi, PhD, Mark Layton, MD, Josu de la Fuente, MRCP, MRCPCH, FRCPath, Irene Roberts, Lucio Luzzatto, MD, FRCP, FRCPath, Rosario Notaro, MD, Anastasios Karadimitris, MD, PhD

Blood (2012) 120 (21): 647.

https://doi.org/10.1182/blood.V120.21.647.647

Published: 2012

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The Association of Anemia and Patient Reported Outcomes in the New Era of PNH Treatment

Factor B Inhibition with Oral Iptacopan Monotherapy Demonstrates Sustained Long-Term Efficacy and Safety in Anti-C5-Treated Patients (pts) with Paroxysmal Nocturnal Hemoglobinuria (PNH) and Persistent Anemia: Final 48-Week Results from the Multicenter, Phase III APPLY-PNH Trial

Ravulizumab Provides Durable Control of Intravascular Hemolysis and Improves Survival in Patients with Paroxysmal Nocturnal Hemoglobinuria: Long-Term Follow-up of Study 301 and Comparisons with Patients of the International PNH Registry

Vemircopan (ALXN2050) Monotherapy in Paroxysmal Nocturnal Hemoglobinuria: Interim Data from a Phase 2 Open-Label Proof-of-Concept Study

Phase 2 study of danicopan in patients with paroxysmal nocturnal hemoglobinuria with an inadequate response to eculizumab

Complement-mediated oxidative damage of red cells impairs response to eculizumab in a G6PD-deficient patient with PNH

COVID-19 Infection in Patients with Paroxysmal Nocturnal Hemoglobinuria in Italy

Efficacy and Safety of Concomitant Use of Ravulizumab and IST in Patients with Paroxysmal Nocturnal Hemoglobinuria up to 52 Weeks

Glucose-6-phosphate dehydrogenase deficiency

A Phase 2 Open-Label Study of Danicopan (ACH-0144471) in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have an Inadequate Response to Eculizumab Monotherapy

Hematological Response to Eculizumab in Paroxysmal Nocturnal Hemoglobinuria: Application of a Novel Classification to Identify Unmet Clinical Needs and Future Clinical Goals

Mechanistic Evaluation of Efficacy Using Biomarkers of the Oral, Small Molecule Factor D Inhibitor, Danicopan (ACH-4471), in Untreated Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)

Two Distinct but Functionally Related Autoreactive T Cell Populations in Paroxysmal Nocturnal Hemoglobinuria and Idiopathic Aplastic Anemia: Glycosylphosphatidylinositol-Specific and IFNγ-Producing T Cells

Glycosylphosphatidylinositol-specific T cells, IFN-γ-producing T cells, and pathogenesis of idiopathic aplastic anemia

CD59 Deficiency Is Critical for C3 Binding on Red Blood Cells of Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) during Anti-C5 Treatment (eculizumab)

Auto-Reactive Glycosyl-Phosphatidyl-Inositol (GPI) Specific T Cells Are Present in Most Patients with Idiopathic Aplastic Anemia (IAA)

Complement Activation in Paroxysmal Nocturnal Hemoglobinuria (PNH) Causes Oxidative Damage Which May Affect Response to Eculizumab

Peptide inhibitors of C3 activation as a novel strategy of complement inhibition for the treatment of paroxysmal nocturnal hemoglobinuria

Glycosylphosphatidylinositol-specific, CD1d-restricted T cells in paroxysmal nocturnal hemoglobinuria

Autoreactive T Cells From Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Specifically Recognize Glycosyl- Phosphatidyl-Inositol (GPI)

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