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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

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

Human Hematopoietic Progenitor Cells and Erythrocytes Generated from Human iPSCs Lacking CD55

Mengyao Wu, Senquan Liu, Hao Bai, PhD, Yongxing Gao, Rachel Evans, David Sullivan, Tong Chen, MD, Linzhao Cheng, PhD

Blood (2017) 130 (Supplement 1): 2204.

https://doi.org/10.1182/blood.V130.Suppl_1.2204.2204

Published: 2017

Journal Articles

Evaluation of Bacteria-Mediated Potential "Bystander" Hemolysis of PNH RED CELLS I n Vitro : NO Evidence of Significant Complement Classical or Lectin Pathway-Mediated Hemolysis Induced by Microorganisms

Xuan Yuan, MD MSc, Guangwei Yang, PhD, Jane A Thanassi, Manuel D Galvan, PhD, Steven D Podos, PhD, Mingjun Huang, PhD, Robert A. Brodsky, MD

Blood (2016) 128 (22): 2431.

https://doi.org/10.1182/blood.V128.22.2431.2431

Published: 2016

Journal Articles

PolyP’s many faces

Alvin H. Schmaier

Blood (2016) 128 (13): 1669–1670.

https://doi.org/10.1182/blood-2016-07-724971

Published: 2016

Journal Articles

A Novel and Pivotal Role of the Mannose-Binding Lectin (MBL) Pathway of Complement Cascade (ComC) Activation in Triggering Mobilization of Hematopoietic Stem/Progenitor Cells (HSPCs)

Mateusz Adamiak, MS, Ahmed Abdel-Latif, MD PhD, Janina Ratajczak, MD PhD, Mariusz Z Ratajczak, MD PhD

Blood (2015) 126 (23): 4301.

https://doi.org/10.1182/blood.V126.23.4301.4301

Published: 2015

Journal Articles

Usefulness of Screening for Paroxismal Nocturnal Hemoglobinuria in Patients with Undefined Anemia

Antonio Giovanni Marino, MD, Esther Natalie Oliva, MD, Bianca Maria Oliva, BSc, Giovanna Sofo, BSc, Natale Ranieri, MD, Francesca Ronco

Blood (2014) 124 (21): 5161.

https://doi.org/10.1182/blood.V124.21.5161.5161

Published: 2014

Journal Articles

Preclinical Evaluation of Orally Bioavailable Small-Molecule Inhibitors of Complement Factor D As a Potential Treatment for Paroxysmal Nocturnal Hemoglobinuria

Jason Wiles, PhD, Steven Podos, PhD, Jane Thanassi, Avinash Phadke, PhD, Venkat Gadhachanda, PhD, Godwin Pais, PhD, Akihiro Hashimoto, PhD, Qiuping Wang, PhD, Dawei Chen, PhD, Xiangzhu Wang, PhD, Atul Agarwal, PhD, Jose Rivera, Michael Elliot, Christopher Marlor, Jay Zhang, Milind Deshpande, PhD, Mingjun Huang, PhD, Yongqing Huang, PhD

Blood (2014) 124 (21): 4819.

https://doi.org/10.1182/blood.V124.21.4819.4819

Published: 2014

Journal Articles

Development Of RNAi Therapeutics Targeting The Complement Pathway

Anna Borodovsky, PhD, Kristina Yucius, Andrew Sprague, James Butler, PhD, Shannon Fishman, BS, Tuyen Nguyen, Akshay Vaishnaw, MD, PhD, Martin Maier, PhD, Rajeev Kallanthottathil, PhD, Satya Kuchimanchi, PhD, Muthiah Manoharan, PhD, Rachel Meyers, PhD, Kevin Fitzgerald, PhD

Blood (2013) 122 (21): 2471.

https://doi.org/10.1182/blood.V122.21.2471.2471

Published: 2013

Journal Articles

A Novel Hybrid CFH/CFHR1–3 Gene in Atypical Hemolytic Uremic Syndrome

Mehmet F. Hepgur, MD, Preeti Chaudhary, MD, Sarmen Sarkissian, MD, Richard J. H. Smith, MD, Howard Liebman, MD, Ilene C Weitz, MD

Blood (2012) 120 (21): 4644.

https://doi.org/10.1182/blood.V120.21.4644.4644

Published: 2012

Journal Articles

Novel Complement Modulators for Paroxysmal Nocturnal Hemoglobinuria: Peptide and Protein Inhibitors of C3 Convertase Prevent Both Surface C3 Deposition and Subsequent Hemolysis of Affected Erythrocytes in Vitro

Antonio M. Risitano, MD, PhD, Patrizia Ricci, Caterina Pascariello, Maddalena Raia, Christoph Q Schmidt, Yingxue Li, Edimara S Reis, Michela Sica, PhD, Rosario Notaro, MD, Luigi Del Vecchio, MD, Fabrizio Pane, Daniel Ricklin, John D Lambris

Blood (2012) 120 (21): 370.

https://doi.org/10.1182/blood.V120.21.370.370

Published: 2012

Journal Articles

Studies in C4b-Deficient Mice Provide Further Evidence That Complement Cascade Orchestrates the Mobilization of Hematopoietic Stem/Progenitor Cells.

Anna Janowska-Wieczorek, MD, PhD, Leah A. Marquez-Curtis, PhD, Danila Leontyev, PhD, Donald R. Branch, PhD, Janina Ratajczak, MD, PhD, Mariusz Z Ratajczak, MD, PhD

Blood (2012) 120 (21): 2316.

https://doi.org/10.1182/blood.V120.21.2316.2316

Published: 2012

Journal Articles

A prevalent C3 mutation in aHUS patients causes a direct C3 convertase gain of function

Lubka T. Roumenina, Marie Frimat, Elizabeth C. Miller, Francois Provot, Marie-Agnes Dragon-Durey, Pauline Bordereau, Sylvain Bigot, Christophe Hue, Simon C. Satchell, Peter W. Mathieson, Christiane Mousson, Christian Noel, Catherine Sautes-Fridman, Lise Halbwachs-Mecarelli, John P. Atkinson, Arnaud Lionet, Veronique Fremeaux-Bacchi

Blood (2012) 119 (18): 4182–4191.

https://doi.org/10.1182/blood-2011-10-383281

Published: 2012

Journal Articles

β 2 -glycoprotein I, the major target in antiphospholipid syndrome, is a special human complement regulator

Katharina Gropp, Nadia Weber, Michael Reuter, Sven Micklisch, Isabell Kopka, Teresia Hallström, Christine Skerka

Blood (2011) 118 (10): 2774–2783.

https://doi.org/10.1182/blood-2011-02-339564

Published: 2011

Includes: Supplemental data

Journal Articles

TT30, a Novel Regulator of the Complement Alternative Pathway (CAP), Inhibits Hemolysis of Paroxysmal Nocturnal Hemoglobinuria (PNH) Erythrocytes and Prevents Upstream C3 Binding On Their Surface in An in Vitro Model.

Antonio M. Risitano, MD, PhD, V. Michael Holers, Bruno Rotoli

Blood (2009) 114 (22): 158.

https://doi.org/10.1182/blood.V114.22.158.158

Published: 2009

Journal Articles

Hyperfunctional C3 convertase leads to complement deposition on endothelial cells and contributes to atypical hemolytic uremic syndrome

Lubka T. Roumenina, Mathieu Jablonski, Christophe Hue, Jacques Blouin, Jordan D. Dimitrov, Marie-Agnes Dragon-Durey, Mathieu Cayla, Wolf H. Fridman, Marie-Alice Macher, David Ribes, Luc Moulonguet, Lionel Rostaing, Simon C. Satchell, Peter W. Mathieson, Catherine Sautes-Fridman, Chantal Loirat, Catherine H. Regnier, Lise Halbwachs-Mecarelli, Veronique Fremeaux-Bacchi

Blood (2009) 114 (13): 2837–2845.

https://doi.org/10.1182/blood-2009-01-197640

Published: 2009

Includes: Supplemental data

Journal Articles

Factor H–related protein 1 (CFHR-1) inhibits complement C5 convertase activity and terminal complex formation

Stefan Heinen, Andrea Hartmann, Nadine Lauer, Ulrike Wiehl, Hans-Martin Dahse, Sylvia Schirmer, Katharina Gropp, Tina Enghardt, Reinhard Wallich, Steffi Hälbich, Michael Mihlan, Ursula Schlötzer-Schrehardt, Peter F. Zipfel, Christine Skerka

Blood (2009) 114 (12): 2439–2447.

https://doi.org/10.1182/blood-2009-02-205641

Published: 2009

Includes: Supplemental data

Journal Articles

Blockade of Intravascular Hemolysis in PNH with the Terminal Complement Inhibitor Eculizumab Unmasks Low-Level Hemolysis Potentially Occurring through C3 Opsonization.

Anita Hill, MD, Russell P. Rother, PhD, Antonio M. Risitano, PhD, Duncan S. Cole, PhD, Matthew J. Cullen, MSc, Stephen J. Richards, PhD, Carmine Selleri, MD, Patrizia Ricci, BSc, Bruno Rotoli, MD, Lucio Luzzatto, PhD, Peter Hillmen, PhD

Blood (2006) 108 (11): 972.

https://doi.org/10.1182/blood.V108.11.972.972

Published: 2006

Journal Articles

Intravenously applied IgG stimulates complement attenuation in a complement-dependent autoimmune disease at the amplifying C3 convertase level

Hans U. Lutz, Pia Stammler, Valentina Bianchi, Ralph M. Trüeb, Thomas Hunziker, Reinhard Burger, Emiliana Jelezarova, Peter J. Späth

Blood (2004) 103 (2): 465–472.

https://doi.org/10.1182/blood-2003-05-1530

Published: 2004

NARROW

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

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)

Human Hematopoietic Progenitor Cells and Erythrocytes Generated from Human iPSCs Lacking CD55

Evaluation of Bacteria-Mediated Potential "Bystander" Hemolysis of PNH RED CELLS I n Vitro : NO Evidence of Significant Complement Classical or Lectin Pathway-Mediated Hemolysis Induced by Microorganisms

PolyP’s many faces

A Novel and Pivotal Role of the Mannose-Binding Lectin (MBL) Pathway of Complement Cascade (ComC) Activation in Triggering Mobilization of Hematopoietic Stem/Progenitor Cells (HSPCs)

Usefulness of Screening for Paroxismal Nocturnal Hemoglobinuria in Patients with Undefined Anemia

Preclinical Evaluation of Orally Bioavailable Small-Molecule Inhibitors of Complement Factor D As a Potential Treatment for Paroxysmal Nocturnal Hemoglobinuria

Development Of RNAi Therapeutics Targeting The Complement Pathway

A Novel Hybrid CFH/CFHR1–3 Gene in Atypical Hemolytic Uremic Syndrome

Novel Complement Modulators for Paroxysmal Nocturnal Hemoglobinuria: Peptide and Protein Inhibitors of C3 Convertase Prevent Both Surface C3 Deposition and Subsequent Hemolysis of Affected Erythrocytes in Vitro

Studies in C4b-Deficient Mice Provide Further Evidence That Complement Cascade Orchestrates the Mobilization of Hematopoietic Stem/Progenitor Cells.

A prevalent C3 mutation in aHUS patients causes a direct C3 convertase gain of function

β 2 -glycoprotein I, the major target in antiphospholipid syndrome, is a special human complement regulator

TT30, a Novel Regulator of the Complement Alternative Pathway (CAP), Inhibits Hemolysis of Paroxysmal Nocturnal Hemoglobinuria (PNH) Erythrocytes and Prevents Upstream C3 Binding On Their Surface in An in Vitro Model.

Hyperfunctional C3 convertase leads to complement deposition on endothelial cells and contributes to atypical hemolytic uremic syndrome

Factor H–related protein 1 (CFHR-1) inhibits complement C5 convertase activity and terminal complex formation

Blockade of Intravascular Hemolysis in PNH with the Terminal Complement Inhibitor Eculizumab Unmasks Low-Level Hemolysis Potentially Occurring through C3 Opsonization.

Intravenously applied IgG stimulates complement attenuation in a complement-dependent autoimmune disease at the amplifying C3 convertase level

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American Society of Hematology

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ASH Publications

American Society of Hematology

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