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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

An Alternative Pathway Specific Flow Cytometric Assay to Detect Complement Activation in Atypical Hemolytic Uremic Syndrome (aHUS)

Jia Yu, Xuan Yuan, MD MSc, Kang Zhou, Daniel White, Samuel A. Merrill, MD PhD, Robert A. Brodsky, MD

Blood (2018) 132 (Supplement 1): 3748.

https://doi.org/10.1182/blood-2018-99-119445

Published: 2018

Journal Articles

Effective Treatment of Thrombotic Microangiopathy Associated with Lupus Nephritis with Eculizumab: a Series of 8 Cases

Mihee Park, MD BS, Ilene Ceil Weitz, MD

Blood (2017) 130 (Supplement 1): 1043.

https://doi.org/10.1182/blood.V130.Suppl_1.1043.1043

Published: 2017

Journal Articles

Three Cases of Patients with Complement Regulatory Factor Genetic Mutations and Acquired Thrombotic Thrombocytopenic Purpura (TTP)

Shebli Atrash, MD, David Kent McKelvey, MD, Appalanaidu Sasapu, MD MS, Muthu Veeraputhiran, MD MPH, Soumya Pandey, MD, Michele H. Cottler-Fox, MD, Pooja Motwani, MD

Blood (2016) 128 (22): 1362.

https://doi.org/10.1182/blood.V128.22.1362.1362

Published: 2016

Journal Articles

Antiphospholipid Syndrome (APS) Patient Plasmas Consistently Activate Complement: A Novel Approach to Detecting a Pathogenic Mechanism for APS

Jacob H. Rand, MD, Xiao-Xuan Wu, MD, Lucia R Wolgast, MD, Victor Lei, MSc, Edward M. Conway, MD PhD

Blood (2016) 128 (22): 719.

https://doi.org/10.1182/blood.V128.22.719.719

Published: 2016

Journal Articles

Small Molecule Factor D Inhibitors Block Complement Activation in Paroxysmal Nocturnal Hemoglobinuria and Atypical Hemolytic Uremic Syndrome

Eleni Gavriilaki, MD, Jane A Thanassi, BS, Guangwei Yang, Xuan Yuan, MD MSc, Mngjun Huang, Robert A. Brodsky, MD

Blood (2015) 126 (23): 275.

https://doi.org/10.1182/blood.V126.23.275.275

Published: 2015

Journal Articles

Mesenchymal Cell Associated Fibrosis in Experimental mpl W515L Mouse Model of Myelofibrosis

Lanzhu Yue, PhD, Ying Han, MD, Ling Zhang, MD, Sheng Wei, MD, Xiubao Ren, MD, Zonghong Shao, Pearlie K Epling-Burnette, PhD

Blood (2015) 126 (23): 604.

https://doi.org/10.1182/blood.V126.23.604.604

Published: 2015

Journal Articles

Novel Genetic Variants in Complement-Mediated Thrombotic Microangiopath

Blood (2015) 126 (23): 1050.

https://doi.org/10.1182/blood.V126.23.1050.1050

Published: 2015

Journal Articles

Von Willebrand Factor Is a Cofactor in Complement Regulation

Shuju Feng, PhD, Michael H. Kroll, MD, Vahid Afshar-Kharghan, MD

Blood (2014) 124 (21): 109.

https://doi.org/10.1182/blood.V124.21.109.109

Published: 2014

Journal Articles

Novel Small-Molecule Inhibitors Targeting Complement Factor D for Therapy of Paroxysmal Nocturnal Hemoglobinuria

B. Paul Morgan, PhD, Jane Thanassi, Steven Podos, PhD, Avinash Phadke, PhD, Venkat Gadhachanda, PhD, Godwin Pais, PhD, Akihiro Hashimoto, PhD, Qiuping Wang, PhD, Dawei Chen, PhD, Xiangzhu Wang, PhD, Atul Agarwal, PhD, Milind Deshpande, PhD, Yongqing Huang, PhD, Jason Wiles, PhD, Mingjun Huang, PhD

Blood (2014) 124 (21): 4817.

https://doi.org/10.1182/blood.V124.21.4817.4817

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

Alternative and Terminal Complement Pathway Biomarkers At Presentation More Precisely Define The Clinical Diagnosis Of aHUS

Spero R Cataland, M.D., V. Michael Holers, M.D., Shangbin Yang, Ph.D., Haifeng M Wu, M.D.

Blood (2013) 122 (21): 3552.

https://doi.org/10.1182/blood.V122.21.3552.3552

Published: 2013

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

Atypical Hemolytic Uremic Syndrome In Japan Characterized By The Inhibitory Antibody-Based Hemolytic Assay and The Gene Analysis

Yoko Yoshida, MS, Xinping Fan, MD, Yoshifumi Ohyama, PhD, Tetsuro Kokubo, PhD, Masanori Matsumoto, MD, PhD, Hideo Yagi, MD, PhD, Hiroko Shirotani-Ikejima, PhD, Toshiyuki Miyata, PhD, Yoshihiro Fujimura, MD, PhD

Blood (2013) 122 (21): 2318.

https://doi.org/10.1182/blood.V122.21.2318.2318

Published: 2013

Journal Articles

The role of complement component 3 (C3) in differentiation of myeloid-derived suppressor cells

Ching-Chuan Hsieh, Hong-Shiue Chou, Horng-Ren Yang, Feng Lin, Sumantha Bhatt, Jie Qin, Lianfu Wang, John J. Fung, Shiguang Qian, Lina Lu

Blood (2013) 121 (10): 1760–1768.

https://doi.org/10.1182/blood-2012-06-440214

Published: 2013

Includes: Supplemental data

Journal Articles

ADAMTS13 Variants in Atypical Hemolytic Uremic Syndrome

Shuju Feng, PhD, Stephen J. Eyler, MD, Yuzhou Zhang, PhD, Tara K. Maga, MD, Michael H. Kroll, MD, Richard J. H. Smith, MD, Vahid Afshar-Kharghan, MD

Blood (2012) 120 (21): 490.

https://doi.org/10.1182/blood.V120.21.490.490

Published: 2012

Journal Articles

Long-Term Outcome in a Pediatric Patient with Atypical Hemolytic Uremic Syndrome (aHUS) with Sustained Eculizumab (ECU) Treatment

Ralph A. Gruppo, MD, Bradley P Dixon, MD

Blood (2011) 118 (21): 4682.

https://doi.org/10.1182/blood.V118.21.4682.4682

Published: 2011

Journal Articles

Efficient osteoclast differentiation requires local complement activation

Zhidan Tu, Hong Bu, James E. Dennis, Feng Lin

Blood (2010) 116 (22): 4456–4463.

https://doi.org/10.1182/blood-2010-01-263590

Published: 2010

Journal Articles

Complement Activation In Patients with Isolated Antiphospholipid Antibodies (aPL) or Primary Antiphospholipid Syndrome (PAPS)

Karen A Breen, MB, Kiran Parmar, MSc, Beverley J Hunt, MD

Blood (2010) 116 (21): 4216.

https://doi.org/10.1182/blood.V116.21.4216.4216

Published: 2010

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)

An Alternative Pathway Specific Flow Cytometric Assay to Detect Complement Activation in Atypical Hemolytic Uremic Syndrome (aHUS)

Effective Treatment of Thrombotic Microangiopathy Associated with Lupus Nephritis with Eculizumab: a Series of 8 Cases

Three Cases of Patients with Complement Regulatory Factor Genetic Mutations and Acquired Thrombotic Thrombocytopenic Purpura (TTP)

Antiphospholipid Syndrome (APS) Patient Plasmas Consistently Activate Complement: A Novel Approach to Detecting a Pathogenic Mechanism for APS

Small Molecule Factor D Inhibitors Block Complement Activation in Paroxysmal Nocturnal Hemoglobinuria and Atypical Hemolytic Uremic Syndrome

Mesenchymal Cell Associated Fibrosis in Experimental mpl W515L Mouse Model of Myelofibrosis

Novel Genetic Variants in Complement-Mediated Thrombotic Microangiopath

Von Willebrand Factor Is a Cofactor in Complement Regulation

Novel Small-Molecule Inhibitors Targeting Complement Factor D for Therapy of Paroxysmal Nocturnal Hemoglobinuria

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

Alternative and Terminal Complement Pathway Biomarkers At Presentation More Precisely Define The Clinical Diagnosis Of aHUS

Development Of RNAi Therapeutics Targeting The Complement Pathway

Atypical Hemolytic Uremic Syndrome In Japan Characterized By The Inhibitory Antibody-Based Hemolytic Assay and The Gene Analysis

The role of complement component 3 (C3) in differentiation of myeloid-derived suppressor cells

ADAMTS13 Variants in Atypical Hemolytic Uremic Syndrome

Long-Term Outcome in a Pediatric Patient with Atypical Hemolytic Uremic Syndrome (aHUS) with Sustained Eculizumab (ECU) Treatment

Efficient osteoclast differentiation requires local complement activation

Complement Activation In Patients with Isolated Antiphospholipid Antibodies (aPL) or Primary Antiphospholipid Syndrome (PAPS)

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American Society of Hematology

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ASH Publications

American Society of Hematology

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