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Journal Articles

Health care resource utilization and cost burden of hemophilia B in the United States

Tyler W. Buckner, Iryna Bocharova, Kaitlin Hagan, Arielle G. Bensimon, Hongbo Yang, Eric Q. Wu, Eileen K. Sawyer, Nanxin Li
Journal: Blood Advances
Blood Adv (2021) 5 (7): 1954–1962.
https://doi.org/10.1182/bloodadvances.2020003424
Published: 2021
Includes: Supplemental data
Journal Articles

Evolutionary insights into coagulation factor IX Padua and other high-specific-activity variants

Benjamin J. Samelson-Jones, Jonathan D. Finn, Leslie J. Raffini, Elizabeth P. Merricks, Rodney M. Camire, Timothy C. Nichols, Valder R. Arruda
Journal: Blood Advances
Blood Adv (2021) 5 (5): 1324–1332.
https://doi.org/10.1182/bloodadvances.2019000405
Published: 2021
Includes: Supplemental data
Journal Articles

Platelet-targeted hyperfunctional FIX gene therapy for hemophilia B mice even with preexisting anti-FIX immunity

Jocelyn A. Schroeder, Juan Chen, Yingyu Chen, Yuanhua Cai, Hongyin Yu, Jeremy G. Mattson, Paul E. Monahan, Qizhen Shi
Journal: Blood Advances
Blood Adv (2021) 5 (5): 1224–1238.
https://doi.org/10.1182/bloodadvances.2020004071
Published: 2021
Includes: Supplemental data
Journal Articles

Concizumab: a novel anti-TFPI therapeutic for hemophilia

Clinical Trials & Observations
Amy D. Shapiro
Journal: Blood Advances
Blood Adv (2021) 5 (1): 279.
https://doi.org/10.1182/bloodadvances.2019001140
Published: 2021
Includes: Supplemental data
Journal Articles

Von Drygalski A, Giermasz A, Castaman G, et al. Etranacogene dezaparvovec (AMT-061 phase 2b): normal/near normal FIX activity and bleed cessation in hemophilia B. Blood Adv. 2019;3(21):3241-3247.

Journal: Blood Advances
Blood Adv (2020) 4 (15): 3668.
https://doi.org/10.1182/bloodadvances.2020002987
Published: 2020
Journal Articles

Characterization of missense mutations in the signal peptide and propeptide of FIX in hemophilia B by a cell-based assay

Wenwen Gao, Yaqi Xu, Hongli Liu, Meng Gao, Qing Cao, Yiyi Wang, Longteng Cui, Rong Huang, Yan Shen, Sanqiang Li, Haiping Yang, Yixiang Chen, Chaokun Li, Haichuan Yu, Weikai Li, Guomin Shen
Journal: Blood Advances
Blood Adv (2020) 4 (15): 3659–3667.
https://doi.org/10.1182/bloodadvances.2020002520
Published: 2020
Includes: Supplemental data
Journal Articles

Etranacogene dezaparvovec (AMT-061 phase 2b): normal/near normal FIX activity and bleed cessation in hemophilia B

Annette Von Drygalski, Adam Giermasz, Giancarlo Castaman, Nigel S. Key, Susan Lattimore, Frank W. G. Leebeek, Wolfgang Miesbach, Michael Recht, Alison Long, Robert Gut, Eileen K. Sawyer, Steven W. Pipe
Journal: Blood Advances
Blood Adv (2019) 3 (21): 3241–3247.
https://doi.org/10.1182/bloodadvances.2019000811
Published: 2019
Includes: Supplemental data
Journal Articles

Immunoadsorption enables successful rAAV5-mediated repeated hepatic gene delivery in nonhuman primates

David Salas, Karin L. Kwikkers, Nerea Zabaleta, Andrea Bazo, Harald Petry, Sander J. van Deventer, Gloria Gonzalez Aseguinolaza, Valerie Ferreira
Journal: Blood Advances
Blood Adv (2019) 3 (17): 2632–2641.
https://doi.org/10.1182/bloodadvances.2019000380
Published: 2019
Includes: Supplemental data
Journal Articles

The frequency of joint hemorrhages and procedures in nonsevere hemophilia A vs B

Clinical Trials & Observations
J. Michael Soucie, Paul E. Monahan, Roshni Kulkarni, Barbara A. Konkle, Marshall A. Mazepa
Journal: Blood Advances
Blood Adv (2018) 2 (16): 2136–2144.
https://doi.org/10.1182/bloodadvances.2018020552
Published: 2018
Journal Articles

Complete correction of hemophilia B phenotype by FIX-Padua skeletal muscle gene therapy in an inhibitor-prone dog model

Robert A. French, Benjamin J. Samelson-Jones, Glenn P. Niemeyer, Clinton D. Lothrop, Jr, Elizabeth P. Merricks, Timothy C. Nichols, Valder R. Arruda
Journal: Blood Advances
Blood Adv (2018) 2 (5): 505–508.
https://doi.org/10.1182/bloodadvances.2017015313
Published: 2018
Includes: Supplemental data
Journal Articles

Enhanced liver gene transfer and evasion of preexisting humoral immunity with exosome-enveloped AAV vectors

Amine Meliani, Florence Boisgerault, Zachary Fitzpatrick, Solenne Marmier, Christian Leborgne, Fanny Collaud, Marcelo Simon Sola, Severine Charles, Giuseppe Ronzitti, Alban Vignaud, Laetitia van Wittenberghe, Beatrice Marolleau, Fabienne Jouen, Sisareuth Tan, Olivier Boyer, Olivier Christophe, Alain R. Brisson, Casey A. Maguire, Federico Mingozzi
Journal: Blood Advances
Blood Adv (2017) 1 (23): 2019–2031.
https://doi.org/10.1182/bloodadvances.2017010181
Published: 2017
Includes: Supplemental data
Journal Articles

Novel approach to genetic analysis and results in 3000 hemophilia patients enrolled in the My Life, Our Future initiative

Clinical Trials & Observations
Jill M. Johnsen, Shelley N. Fletcher, Haley Huston, Sarah Roberge, Beth K. Martin, Martin Kircher, Neil C. Josephson, Jay Shendure, Sarah Ruuska, Marion A. Koerper, Jaime Morales, Glenn F. Pierce, Diane J. Aschman, Barbara A. Konkle
Journal: Blood Advances
Blood Adv (2017) 1 (13): 824–834.
https://doi.org/10.1182/bloodadvances.2016002923
Published: 2017
Includes: Supplemental data