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Journal Articles

Gene therapy for hemophilia B using CB 2679d-GT: a novel factor IX variant with higher potency than factor IX Padua

Nisha Nair, Dries De Wolf, Phuong Anh Nguyen, Quang Hong Pham, Ermira Samara-Kuko, Jeff Landau, Grant E. Blouse, Marinee K. Chuah, Thierry VandenDriessche

Blood (2021) 137 (21): 2902–2906.

https://doi.org/10.1182/blood.2020006005

Published: 2021

Includes: Supplemental data

Journal Articles

Red blood cell microvesicles activate the contact system, leading to factor IX activation via 2 independent pathways

Denis F. Noubouossie, Michael W. Henderson, Micah Mooberry, Anton Ilich, Patrick Ellsworth, Mark Piegore, Sarah C. Skinner, Rafal Pawlinski, Ian Welsby, Thomas Renné, Maureane Hoffman, Dougald M. Monroe, Nigel S. Key

Blood (2020) 135 (10): 755–765.

https://doi.org/10.1182/blood.2019001643

Published: 2020

Includes: Supplemental data

Journal Articles

Long-Term AAV-Mediated Factor VIII Expression in Nine Hemophilia A Dogs: A 10 Year Follow-up Analysis on Durability, Safety and Vector Integration

Giang N. Nguyen, BS, John K. Everett, PhD, Hayley Raymond, BA, Samita Kafle, BS, Elizabeth P. Merricks, PhD, Haig H. Kazazian, MD, Timothy C. Nichols, MD, Frederic D. Bushman, PhD, Denise E. Sabatino, PhD

Blood (2019) 134 (Supplement_1): 611.

https://doi.org/10.1182/blood-2019-126007

Published: 2019

Journal Articles

Stable FIX Expression and Durable Reductions in Bleeding and Factor IX Consumption for up to 4 Years Following AMT-060 Gene Therapy in Adults with Severe or Moderate-Severe Hemophilia B

Wolfgang Miesbach, MD PhD, Karina Meijer, Michiel Coppens, MD, Peter Kampmann, MD, Dr. Klamroth, Roger Schutgens, MD PhD MSc, Giancarlo Castaman, MD, Erhard Seifried, MD PhD, Joachim Schwaeble, Halvard Bönig, Eileen K Sawyer, PhD, Frank W.G. Leebeek, MD PhD

Blood (2019) 134 (Supplement_1): 2059.

https://doi.org/10.1182/blood-2019-122535

Published: 2019

Journal Articles

Prophylactic Treatment in People with Severe Hemophilia B in the US: An Analysis of Real-World Healthcare System Costs and Clinical Outcomes

Declan Noone, MSc, Gabriel Pedra, PhD, Sohaib Asghar, MSc, Jamie O'Hara, MSc, Eileen K Sawyer, PhD, Nanxin (Nick) Li, PhD

Blood (2019) 134 (Supplement_1): 2118.

https://doi.org/10.1182/blood-2019-126576

Published: 2019

Journal Articles

Laminin G Domains of Protein S Bind and Inhibit Fixa

Sabyasachi Chatterjee, PhD MSc, BSc, Amber Sylvain, Undergraduate Student, Satish Pilli, PhD, Whitney Fant, MS, Doerte R. Fricke, MS, Terry Watt, PhD, Rinku Majumder, PhD

Blood (2019) 134 (Supplement_1): 3624.

https://doi.org/10.1182/blood-2019-127112

Published: 2019

Journal Articles

Combined Point of Care Tools Are Able to Improve Treatment Adherence and Health-Related Quality of Life in Patients with Severe Hemophilia: An Observational Prospective Study

Mariasanta Napolitano, Simona Raso, MD, Maria Francesca Mansueto, PhD, Salvatrice Mancuso, MD, Matteo Nicola Dario Di Minno, MD PhD, Dalila Scaturro, MD, Giulia Letizia Mauro, Prof,MD, Giovanni Di Minno, Sergio Siragusa, Prof, MD

Blood (2019) 134 (Supplement_1): 3455.

https://doi.org/10.1182/blood-2019-128129

Published: 2019

Journal Articles

Evaluation of the Human Factor IX Gene Therapy Vector TAK-748 in Hemophilia: Results from Non-Clinical Studies in Factor IX Knockout Mice and Rhesus Monkeys

Markus Weiller, Helen Wang, Sogué Coulibaly, Maria Schuster, Hanspeter Rottensteiner, Kefeng Sun, Marinee K Chuah, Thierry Vandendriessche, Friedrich Scheiflinger, Werner Höllriegl

Blood (2019) 134 (Supplement_1): 4633.

https://doi.org/10.1182/blood-2019-124734

Published: 2019

Journal Articles

Spontaneous Bleeding and Poor Bleeding Response with Extended Half-Life Factor IX Products: A Survey of Select US and Canadian Hemophilia Treatment Centers

Lynn M Malec, MD, Stacy E. Croteau, Michael Callaghan, MD, Davide Matino, MD MSc, Kenneth Dale Friedman, MD, Robert F. Sidonio, Jr., MD MSc

Blood (2019) 134 (Supplement_1): 2407.

https://doi.org/10.1182/blood-2019-126196

Published: 2019

Journal Articles

Plasma Kallikrein Contributes to Ellagic Acid-Induced Coagulation in the Absence of FXI By Activating Factor IX

Mayken Visser, Rene van Oerle, Hugo ten Cate, MD PhD, Volker Laux, PhD, Stefan Heitmeier, PhD, Henri M.H. Spronk, PhD

Blood (2019) 134 (Supplement_1): 1106.

https://doi.org/10.1182/blood-2019-131738

Published: 2019

Journal Articles

Evaluation of a Blood Coagulation Factor IX Variant That Functions Independently of Factor VIII As an Alternative Treatment for Hemophilia A

Viola J.F. Strijbis, MSc, Ka Lei Cheung, Pavlina Konstantinova, PhD, Ying Poi Liu, PhD, Sander J van Deventer, MD PhD, Mettine H.A. Bos, PhD

Blood (2019) 134 (Supplement_1): 1110.

https://doi.org/10.1182/blood-2019-124120

Published: 2019

Journal Articles

Toll-like Receptor 9 Activation Accelerates Inhibitor Formation in Response to Factor VIII

Radoslaw Kaczmarek, Alexandra Sherman, DVM, Moanaro Biswas, PhD, Roland W Herzog, PhD

Blood (2019) 134 (Supplement_1): 1113.

https://doi.org/10.1182/blood-2019-125348

Published: 2019

Journal Articles

Lack of Inhibitor Development in the American Thrombosis and Hemostasis Network (ATHN)-2 Factor Switching Study: Preliminary Report of Primary Outcome

Robert F. Sidonio, Jr., MD MSc, Dunlei Cheng, PhD, Christine Guelcher, PPCNP-BC, Janna M. Journeycake, MD, Susan U Lattimore, Clifford Takemoto, MD, Michael D. Tarantino, MD, Crystal Watson, Ellis J. Neufeld, MD, Carol Fedor, RN

Blood (2019) 134 (Supplement_1): 1114.

https://doi.org/10.1182/blood-2019-128612

Published: 2019

Journal Articles

Evaluation of the in Vitro Procoagulant Effect of Factor IX Concentrates in Patients on Prophylaxis with Emicizumab

Ihosvany Fernandez-Bello, María Teresa Alvarez Román, Mónica Martín, María Isabel Rivas Pollmar, Sara García Barcenilla, Carmen García Martínez, Tamara Cebanu, Paula Acuña, Raul Justo Sanz, Elena Monzón Manzano, Miguel A Canales, MD PhD, Nora Butta, PhD, Victor Jimenez-Yuste

Blood (2019) 134 (Supplement_1): 1118.

https://doi.org/10.1182/blood-2019-129983

Published: 2019

Journal Articles

Thrombin: A Driver or Attenuator of Bone Disease on Hemophilia?

Galen Goldscheitter, BS, BA, Robert Klein, MD, Jason A Taylor, MD PhD

Blood (2019) 134 (Supplement_1): 4916.

https://doi.org/10.1182/blood-2019-130698

Published: 2019

Journal Articles

Immediate and Long-Term Outcomes Following Orthopedic Procedures in Patients with Hemophilia: A Three-Decade Retrospective Review

CHU Yang Lin, MD, Haowei Sun, MD

Blood (2019) 134 (Supplement_1): 5874.

https://doi.org/10.1182/blood-2019-123207

Published: 2019

Journal Articles

Adeno-Associated Virus Mediated Delivery of a TFPI Antibody Restores Hemostasis in Mouse Model of Hemophilia

Swapnil Rakhe, MS, Reema Jasuja, PhD, Rajani Shelke, Anagha Sawant, Suryanarayan Somanathan, PhD, John E. Murphy, PhD, Debra D Pittman, MS

Blood (2019) 134 (Supplement_1): 3357.

https://doi.org/10.1182/blood-2019-130054

Published: 2019

Journal Articles

A CRISPR/Cas9-Generated Murine Model Reveals Cooperation between BCR Signaling and CDKN2A/2B and TP53 Disruption in Richter Syndrome

Supriya Chakraborty, PhD, Ilaria Fortunati, BS, Claudio Martines, MS, Brijesh Yadav, PhD, Marija Dimishkovska, PhD, Idanna Innocenti, MD PhD, Rosa Fazio, BS, Giovanni D'Arena, MD, Aleksandar Dimovski, MD PhD, Luca Laurenti, MD, Dimitar G Efremov, MD PhD

Blood (2019) 134 (Supplement_1): 4278.

https://doi.org/10.1182/blood-2019-128425

Published: 2019

Journal Articles

In Vitro and In Vivo Characterization of Marstacimab, an Anti-TFPI Antibody

Chuenlei Parng, PhD, Macy Jin, PhD, Matthew Holsti, PhD, Susan Benard, Swapnil Rakhe, MS, Sunita R. Patel-Hett, PhD, Alison Joyce, Rob Webster, Debra D Pittman, MS

Blood (2019) 134 (Supplement_1): 2391.

https://doi.org/10.1182/blood-2019-130327

Published: 2019

Journal Articles

Efficacy and Safety in 15 Hemophilia B Patients Treated with the AAV Gene Therapy Vector Fidanacogene Elaparvovec and Followed for at Least 1 Year

Lindsey A. George, MD, Spencer K. Sullivan, MD, John E.J. Rasko, MBBS, PhD, Adam Giermasz, MD PhD, Benjamin J. Samelson-Jones, MD PhD, Jonathan M. Ducore, MD MPH, Jerome M. Teitel, MD FRCPC, Catherine E. McGuinn, MD, Alexa R. Runowski, MS, Fraser Wright, PhD, Xavier M. Anguela, PhD, Katherine A. High, MD, Denis Rybin, PhD, John E. Murphy, PhD, Jeremy Rupon, MD

Blood (2019) 134 (Supplement_1): 3347.

https://doi.org/10.1182/blood-2019-124091

Published: 2019

NARROW

Gene therapy for hemophilia B using CB 2679d-GT: a novel factor IX variant with higher potency than factor IX Padua

Red blood cell microvesicles activate the contact system, leading to factor IX activation via 2 independent pathways

Long-Term AAV-Mediated Factor VIII Expression in Nine Hemophilia A Dogs: A 10 Year Follow-up Analysis on Durability, Safety and Vector Integration

Stable FIX Expression and Durable Reductions in Bleeding and Factor IX Consumption for up to 4 Years Following AMT-060 Gene Therapy in Adults with Severe or Moderate-Severe Hemophilia B

Prophylactic Treatment in People with Severe Hemophilia B in the US: An Analysis of Real-World Healthcare System Costs and Clinical Outcomes

Laminin G Domains of Protein S Bind and Inhibit Fixa

Combined Point of Care Tools Are Able to Improve Treatment Adherence and Health-Related Quality of Life in Patients with Severe Hemophilia: An Observational Prospective Study

Evaluation of the Human Factor IX Gene Therapy Vector TAK-748 in Hemophilia: Results from Non-Clinical Studies in Factor IX Knockout Mice and Rhesus Monkeys

Spontaneous Bleeding and Poor Bleeding Response with Extended Half-Life Factor IX Products: A Survey of Select US and Canadian Hemophilia Treatment Centers

Plasma Kallikrein Contributes to Ellagic Acid-Induced Coagulation in the Absence of FXI By Activating Factor IX

Evaluation of a Blood Coagulation Factor IX Variant That Functions Independently of Factor VIII As an Alternative Treatment for Hemophilia A

Toll-like Receptor 9 Activation Accelerates Inhibitor Formation in Response to Factor VIII

Lack of Inhibitor Development in the American Thrombosis and Hemostasis Network (ATHN)-2 Factor Switching Study: Preliminary Report of Primary Outcome

Evaluation of the in Vitro Procoagulant Effect of Factor IX Concentrates in Patients on Prophylaxis with Emicizumab

Thrombin: A Driver or Attenuator of Bone Disease on Hemophilia?

Immediate and Long-Term Outcomes Following Orthopedic Procedures in Patients with Hemophilia: A Three-Decade Retrospective Review

Adeno-Associated Virus Mediated Delivery of a TFPI Antibody Restores Hemostasis in Mouse Model of Hemophilia

A CRISPR/Cas9-Generated Murine Model Reveals Cooperation between BCR Signaling and CDKN2A/2B and TP53 Disruption in Richter Syndrome

In Vitro and In Vivo Characterization of Marstacimab, an Anti-TFPI Antibody

Efficacy and Safety in 15 Hemophilia B Patients Treated with the AAV Gene Therapy Vector Fidanacogene Elaparvovec and Followed for at Least 1 Year

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American Society of Hematology

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ASH Publications

American Society of Hematology

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