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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

Synergistic Effect of Carfilzomib and Metformin in Vascular Plasticity; The Emerging Role of Autophagy

Panagiotis Efentakis, PhD, Ioanna Andreadou, PhD, Panagiota-Efstathia Nikolaou, PhD, Claudius Witzler, Svenja Siemer, Eftathios Kastritis, Philip Wenzel, MD PhD, Meletios A. Dimopoulos, MD, Evangelos Terpos, MD PhD

Blood (2019) 134 (Supplement_1): 3797.

https://doi.org/10.1182/blood-2019-123522

Published: 2019

Journal Articles

Comparative Evaluation of Complement Factor D, C3, and C5 Inhibitors on Serum Bactericidal Activity Against Neisseria Meningitidis Isolates

Yongsen Zhao, Dharaben Patel, MS, Jane A Thanassi, BS, Mingjun Huang, PhD, Steven D Podos, PhD

Blood (2019) 134 (Supplement_1): 5763.

https://doi.org/10.1182/blood-2019-132253

Published: 2019

Journal Articles

3D-Tissue Engineered Bone Marrow (3DTEBM) Culture Retrospectively Predicts Treatment Clinical Outcomes of Multiple Myeloma Patients

Amanda Jeske, BS, Feda Azab, BPharm, Pilar De La Puente, PhD, Barbara Muz, PhDMSc, Justin King, Daniel R. Kohnen, MBA, Ravi Vij, MBBS, Abdel Kareem Azab, PhD

Blood (2018) 132 (Supplement 1): 1987.

https://doi.org/10.1182/blood-2018-99-118888

Published: 2018

Journal Articles

Overcoming Drug Resistance in Myeloma By Synchronized Delivery of Therapeutic and Bone Marrow Disrupting Agents By Nanoparticles Targeting Tumor-Associated Endothelium

Cinzia Federico, PhD, Barbara Muz, PhDMSc, Jennifer Sun, BSc, Kinan Alhallak, BSc, Justin King, Daniel R. Kohnen, MBA, Mark A. Fiala, BS, Aldo M. Roccaro, MDPhD, Ravi Vij, MBBS, Abdel Kareem Azab, PhD

Blood (2018) 132 (Supplement 1): 1931.

https://doi.org/10.1182/blood-2018-99-118251

Published: 2018

Journal Articles

Minimal Residual Disease (MRD) Analysis Using Multiparametric Flow Cytometry (MFC) and Identification of Differences in Subpopulations Using Next Generation Sequencing (NGS) in the Bone Marrow (BM) of Multiple Myeloma (MM) Patients (pts)

Sandra M. Dold, MSc, BSc, Veronika Riebl, Dagmar Wider, Marie Follo, PhD, Stefan J. Mueller, Ingrid Bartsch, Milena Pantic, PhD, Cornelius Miething, MD, Chrissoula Kiote-Schmidt, K. Martin Kortüm, MD, Mihaela Zlei, Abdel Kareem Azab, PhD, Mascha Binder, MD, Justyna Rawluk, Justus Duyster, Prof., Robert Zeiser, MD, Ralph Wäsch, MD, Monika Engelhardt, MD

Blood (2018) 132 (Supplement 1): 3222.

https://doi.org/10.1182/blood-2018-99-112489

Published: 2018

Journal Articles

In Vitro Combination Studies of ACH-4471 with Eculizumab to Assess a Potential "Switch" Treatment Approach for Paroxysmal Nocturnal Hemoglobinuria

Dharaben Patel, MS, Jane A Thanassi, BS, Guangwei Yang, PhD, Xuan Yuan, MD MSc, Robert A. Brodsky, MD, Mingjun Huang, PhD, Steven D Podos, PhD

Blood (2017) 130 (Supplement 1): 2198.

https://doi.org/10.1182/blood.V130.Suppl_1.2198.2198

Published: 2017

Journal Articles

Lack of an Alberta Newborn Screening Program Is Associated with Delayed Diagnosis of Sickle Cell Disease

Dania Monagel, MDFRCPC,FAAP, Michael Leaker, MDFRCPC, Doan Le, MDFRCPC,FAAP, Nicola A. Wright, MSc, MD FRCPC, Angela Ruzycki, MacGregor Steele, MD FRCPC

Blood (2017) 130 (Supplement 1): 2255.

https://doi.org/10.1182/blood.V130.Suppl_1.2255.2255

Published: 2017

Journal Articles

Eculizumab treatment and impaired opsonophagocytic killing of meningococci by whole blood from immunized adults

Monica Konar, Dan M. Granoff

Blood (2017) 130 (7): 891–899.

https://doi.org/10.1182/blood-2017-05-781450

Published: 2017

Includes: Supplemental data

Journal Articles

Hydroxyurea to Treat Pediatric Sickle Cell Disease in Haiti - a Preliminary Report

Nicholas McGregor, MD, Emmeline Lerebours, MD, Prasad Bodas, MD

Blood (2016) 128 (22): 1313.

https://doi.org/10.1182/blood.V128.22.1313.1313

Published: 2016

Journal Articles

Effect of Complement Inhibition By Anti-C5 (Eculizumab) or a Small Molecule Inhibitor of Factor D (ACH-4471) on Survival of Meningococci in Blood from Vaccinated Adults

Monica Konar, PhD, Eduardo Lujan, Dan Granoff, MD

Blood (2016) 128 (22): 2429.

https://doi.org/10.1182/blood.V128.22.2429.2429

Published: 2016

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

A Novel HIV Envelope Bi-Specific Killer Engager Enhances Natural Killer Cell Mediated ADCC Responses Against HIV-Infected Cells

Zachary B. Davis, PhD BS, Todd Lenvik, Louis Hansen, Martin Felices, PhD, Sarah Cooley, MD, Dan Vallera, PhD, Ashley T. Haase, MD, Richard A. Koup, MD, Daniel Douek, MD, Timothy Schacker, MD, Jeffrey S. Miller, MD

Blood (2016) 128 (22): 2517.

https://doi.org/10.1182/blood.V128.22.2517.2517

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

Integrating Structural and Functional Studies Leads to a New Model of β-Globin Activation That Suggests Distinct Initiation and Maintenance States

M. A. Bender, MD, PhD, Davide Bau', PhD, Tobias Ragoczy, PhD, Rachel Byron, BS, Ye Zhan, BS, Bryan Lajoie, BS, Marc A. Marti-Renom, PhD, Job Dekker, PhD, Mark T. Groudine, MD, PhD

Blood (2011) 118 (21): 349.

https://doi.org/10.1182/blood.V118.21.349.349

Published: 2011

Journal Articles

HIV-1 Tat and heparan sulfate proteoglycan interaction: a novel mechanism of lymphocyte adhesion and migration across the endothelium

Chiara Urbinati, Stefania Nicoli, Mauro Giacca, Guido David, Simona Fiorentini, Arnaldo Caruso, Massimo Alfano, Luca Cassetta, Marco Presta, Marco Rusnati

Blood (2009) 114 (15): 3335–3342.

https://doi.org/10.1182/blood-2009-01-198945

Published: 2009

Includes: Supplemental data

Journal Articles

Ikaros Drives Human Haemoglobin Switching by Facilitating Active Chromatin Hub Formation.

Andrew C. Perkins, MBBS, PhD, Michael Tallack, BSc (Hons), Ye Zhan, PhD, Peter Papathanasiou, BSc, PhD, Crhistopher Goodnow, BSc, PhD, Karin Gaensler, MD, PhD, Paul M. Crossley, BSc, PhD, Job Dekker, BSc, PhD, Janelle R. Keys, BSc, PhD

Blood (2007) 110 (11): 1772.

https://doi.org/10.1182/blood.V110.11.1772.1772

Published: 2007

Journal Articles

Response to Anti CD20 Monoclonal Antibody Rituximab® and Epitope Mapping of Inhibitory Antibodies in Patients with Acquired Haemophilia.

Manuela Krause, MD, Christoph Koenigs, MD, Christoph Kessel, Inge Scharrer, MD, Ralf Grossmann, MD, Wolfhard Kreuz, MD

Blood (2006) 108 (11): 1044.

https://doi.org/10.1182/blood.V108.11.1044.1044

Published: 2006

Journal Articles

D-4F Restores eNOS-Dependent Vasodilation in Sickle Cell Disease by Decreasing Proinflammatory HDL.

Kirkwood A. Pritchard, Jr., Jingli Wang, Hao Xu, Deron W. Jones, Sandra L. Holzhauer, Thomas D. Foster, Keith T. Oldham, Cheryl A. Hillery

Blood (2005) 106 (11): 2324.

https://doi.org/10.1182/blood.V106.11.2324.2324

Published: 2005

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)

Synergistic Effect of Carfilzomib and Metformin in Vascular Plasticity; The Emerging Role of Autophagy

Comparative Evaluation of Complement Factor D, C3, and C5 Inhibitors on Serum Bactericidal Activity Against Neisseria Meningitidis Isolates

3D-Tissue Engineered Bone Marrow (3DTEBM) Culture Retrospectively Predicts Treatment Clinical Outcomes of Multiple Myeloma Patients

Overcoming Drug Resistance in Myeloma By Synchronized Delivery of Therapeutic and Bone Marrow Disrupting Agents By Nanoparticles Targeting Tumor-Associated Endothelium

Minimal Residual Disease (MRD) Analysis Using Multiparametric Flow Cytometry (MFC) and Identification of Differences in Subpopulations Using Next Generation Sequencing (NGS) in the Bone Marrow (BM) of Multiple Myeloma (MM) Patients (pts)

In Vitro Combination Studies of ACH-4471 with Eculizumab to Assess a Potential "Switch" Treatment Approach for Paroxysmal Nocturnal Hemoglobinuria

Lack of an Alberta Newborn Screening Program Is Associated with Delayed Diagnosis of Sickle Cell Disease

Eculizumab treatment and impaired opsonophagocytic killing of meningococci by whole blood from immunized adults

Hydroxyurea to Treat Pediatric Sickle Cell Disease in Haiti - a Preliminary Report

Effect of Complement Inhibition By Anti-C5 (Eculizumab) or a Small Molecule Inhibitor of Factor D (ACH-4471) on Survival of Meningococci in Blood from Vaccinated Adults

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

A Novel HIV Envelope Bi-Specific Killer Engager Enhances Natural Killer Cell Mediated ADCC Responses Against HIV-Infected Cells

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

Integrating Structural and Functional Studies Leads to a New Model of β-Globin Activation That Suggests Distinct Initiation and Maintenance States

HIV-1 Tat and heparan sulfate proteoglycan interaction: a novel mechanism of lymphocyte adhesion and migration across the endothelium

Ikaros Drives Human Haemoglobin Switching by Facilitating Active Chromatin Hub Formation.

Response to Anti CD20 Monoclonal Antibody Rituximab® and Epitope Mapping of Inhibitory Antibodies in Patients with Acquired Haemophilia.

D-4F Restores eNOS-Dependent Vasodilation in Sickle Cell Disease by Decreasing Proinflammatory HDL.

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American Society of Hematology

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American Society of Hematology

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