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Journal Articles

Improved Joint Health in the Phase 3 HOPE-B Trial of Etranacogene Dezaparvovec Gene Therapy for Hemophilia B over 36 Months of Follow up

Rebecca Kruse-Jarres, Bruce Ewenstein, Paul E. Monahan, Nicholas Galante, Nathalie W.D. Jansen, Sean Gill, Niamh O'Connell

Blood (2024) 144 (Supplement 1): 3580.

https://doi.org/10.1182/blood-2024-201395

Published: 2024

Journal Articles

Stable Factor IX Expression and Sustained Reductions in Factor IX Use 8 Years after Gene Therapy with CSL220 (Formerly AMT-060) in Adults with Hemophilia B

Frank W.G. Leebeek, Karina Meijer, Michiel Coppens, Peter Kampmann, Dr. Klamroth, Paul van der Valk, Paul E. Monahan, Karen Pinachyan, Sandra LeQuellec, Wolfgang Miesbach

Blood (2024) 144 (Supplement 1): 3578.

https://doi.org/10.1182/blood-2024-198379

Published: 2024

Journal Articles

Durability of Factor IX Activity and Bleeding Rate in People with Severe or Moderately Severe Hemophilia B after 5 Years of Follow-up in the Phase 1/2 Study of AMT-060, and after 3 Years of Follow-up in the Phase 2b and 2 Years of Follow-up in the Phase 3 Studies of Etranacogene Dezaparvovec (AMT-061)

Wolfgang A. Miesbach, Michael Recht, Nigel S. Key, Krupa Sivamurthy, Paul E. Monahan, Steven W. Pipe

Blood (2022) 140 (Supplement 1): 4913–4914.

https://doi.org/10.1182/blood-2022-166810

Published: 2022

Journal Articles

Adults with Severe or Moderately Severe Hemophilia B Receiving Etranacogene Dezaparvovec in the HOPE-B Phase 3 Clinical Trial Continue to Experience a Stable Increase in Mean Factor IX Activity Levels and Durable Hemostatic Protection after 24 Months’ Follow-up

Steven W. Pipe, Frank W.G. Leebeek, Michael Recht, Nigel S. Key, Susan Lattimore, Giancarlo Castaman, Michiel Coppens, David Cooper, Robert Gut, Sergio Slawka, Stephanie Verweij, Ricardo Dolmetsch, Yanyan Li, Paul E. Monahan, Wolfgang A. Miesbach

Blood (2022) 140 (Supplement 1): 4910–4912.

https://doi.org/10.1182/blood-2022-166135

Published: 2022

Journal Articles

Durability of Bleeding Protection and Factor IX Activity Levels Are Demonstrated in Individuals with and without Adeno-Associated Virus Serotype 5 Neutralizing Antibodies (Titers <1:700) with Comparable Safety in the Phase 3 HOPE-B Clinical Trial of Etranacogene Dezaparvovec Gene Therapy for Hemophilia B

Steven W. Pipe, Frank W.G. Leebeek, Michael Recht, Nigel S. Key, Susan Lattimore, Giancarlo Castaman, David Cooper, Stephanie Verweij, Ricardo Dolmetsch, Jacqueline Tarrant, Yanyan Li, Paul E. Monahan, Wolfgang A. Miesbach

Blood (2022) 140 (Supplement 1): 4904–4906.

https://doi.org/10.1182/blood-2022-166745

Published: 2022

Journal Articles

BAX 335 hemophilia B gene therapy clinical trial results: potential impact of CpG sequences on gene expression

Barbara A. Konkle, Christopher E. Walsh, Miguel A. Escobar, Neil C. Josephson, Guy Young, Annette von Drygalski, Scott W. J. McPhee, R. Jude Samulski, Ivan Bilic, Maurus de la Rosa, Birgit M. Reipert, Hanspeter Rottensteiner, Friedrich Scheiflinger, John C. Chapin, Bruce Ewenstein, Paul E. Monahan

Blood (2021) 137 (6): 763–774.

https://doi.org/10.1182/blood.2019004625

Published: 2021

Includes: Supplemental data

Journal Articles

Abnormal joint and bone wound healing in hemophilia mice is improved by extending factor IX activity after hemarthrosis

Junjiang Sun, Baolai Hua, Eric W. Livingston, Sarah Taves, Peter B. Johansen, Maureane Hoffman, Mirella Ezban, Dougald M. Monroe, Ted A. Bateman, Paul E. Monahan

Blood (2017) 129 (15): 2161–2171.

https://doi.org/10.1182/blood-2016-08-734053

Published: 2017

Includes: Supplemental data

Journal Articles

Platelet-Derived Codon-Optimized Hyperfunctional FIX Gene Therapy of Hemophilia B Mice

Jocelyn A. Schroeder, Mary A. Jozwiak, Paul E. Monahan, MD, Qizhen Shi, MD PhD

Blood (2016) 128 (22): 3751.

https://doi.org/10.1182/blood.V128.22.3751.3751

Published: 2016

Journal Articles

Prophylactic efficacy of BeneFIX vs Alprolix in hemophilia B mice

Brian Cooley, William Funkhouser, Dougald Monroe, Ashley Ezzell, David M. Mann, Feng-Chang Lin, Paul E. Monahan, Darrel W. Stafford

Blood (2016) 128 (2): 286–292.

https://doi.org/10.1182/blood-2016-01-696104

Published: 2016

Journal Articles

Men with severe hemophilia in the United States: birth cohort analysis of a large national database

Marshall A. Mazepa, Paul E. Monahan, Judith R. Baker, Brenda K. Riske, J. Michael Soucie

Blood (2016) 127 (24): 3073–3081.

https://doi.org/10.1182/blood-2015-10-675140

Published: 2016

Includes: Supplemental data

Journal Articles

At Equal Doses FIXFc (Alprolix) Is Not Significanly Better in a Saphenous Vein Bleeding Model Than Recombinant FIX (BeneFix) Seven Days Post-Infusion

Brian Cooley, PhD, David M Mann, PhD, Dougald Monroe, PhD, Feng-Chang Lin, Paul E. Monahan, MD, Darrel W. Stafford, PhD

Blood (2015) 126 (23): 3490.

https://doi.org/10.1182/blood.V126.23.3490.3490

Published: 2015

Journal Articles

The Effect of Prophylactic Recombinant Factor IX and Glycopegylated Factor IX to Normalize Articular Wound Healing Following Hemarthrosis Examined in Hemophilia B Mice

Junjiang Sun, MD, Eric Livingston, Tom Knudsen, DVM, PhD, Dougald M. Monroe, III, PhD, Mirella Ezban, PhD, Ted Bateman, PhD, Paul E. Monahan, MD

Blood (2015) 126 (23): 549.

https://doi.org/10.1182/blood.V126.23.549.549

Published: 2015

Journal Articles

Adeno-Associated Virus 8 (AAV8) Vector Genome Biodistribution into Body Fluids Following Single Intravenous Administration of BAX 335 Gene Therapy for Hemophilia B

Boyan Litchev, MD, Paul E. Monahan, MD, Anne Prener

Blood (2015) 126 (23): 2040.

https://doi.org/10.1182/blood.V126.23.2040.2040

Published: 2015

Journal Articles

Hemophilia B Mouse Lines Doubly Humanized for Human F9 and MHC Class II Genes Advance Specificity of Inhibitor Study

Genlin Hu, PhD, Tong Gui, PhD, Junjiang Sun, MD, Darrel W. Stafford, PhD, R. Jude Samulski, PhD, Paul E. Monahan, MD

Blood (2011) 118 (21): 200.

https://doi.org/10.1182/blood.V118.21.200.200

Published: 2011

Journal Articles

Continued Transmission of Parvovirus B19 in Plasma-Derived Factor Concentrates After the Implementation of B19 Nucleic Acid Plasma Minipool Screening,

J. Michael Soucie, Ph.D., Christine De Staercke, PhD, Meera B. Chitlur, MD, Ralph A. Gruppo, MD, W Craig Hooper, PhD, Craig M. Kessler, MD, Roshni Kulkarni, MD, Marilyn J. Manco-Johnson, MD, Paul E. Monahan, MD, Meredith Oakley, MPH, Jerry Powell, MD, Meredith Pyle, MS, Michael Recht, MD, PhD, Brenda Riske, MS, MBA, MPA, Hernan Sabio, MD, Sean Trimble, MPH

Blood (2011) 118 (21): 3378.

https://doi.org/10.1182/blood.V118.21.3378.3378

Published: 2011

Journal Articles

Employing Factor IX Variants to Avoid Limitations Imposed by Immune Recognition of AAV Vector in Hemophilia B Gene Therapy

Paul E. Monahan, MD, Junjiang Sun, MD, Tong Gui, PhD, David G Wichlan, Scott W McPhee, R. Jude Samulski

Blood (2011) 118 (21): 3124.

https://doi.org/10.1182/blood.V118.21.3124.3124

Published: 2011

Journal Articles

Intraarticular factor IX protein or gene replacement protects against development of hemophilic synovitis in the absence of circulating factor IX

Junjiang Sun, Narine Hakobyan, Leonard A. Valentino, Brian L. Feldman, R. Jude Samulski, Paul E. Monahan

Blood (2008) 112 (12): 4532–4541.

https://doi.org/10.1182/blood-2008-01-131417

Published: 2008

Includes: Supplemental data

Journal Articles

Investigator-Prescribed Prophylaxis of rFIX (BeneFIX ® ) in Children <6 Years of Age: Efficacy and Safety Outcomes.

Paul E. Monahan, MD, Raina Liesner, MD, Sharon Sullivan, PhD, Brooke Hayward, SM, Maria E. Ramirez, PhD, MPH, Patrick Kelly, MD, David A. Roth, MD

Blood (2008) 112 (11): 1221.

https://doi.org/10.1182/blood.V112.11.1221.1221

Published: 2008

Journal Articles

Extravascular Clotting Factor Activity within Joint Tissues Protects Against Progression of Hemophilic Arthropathy.

Junjiang Sun, MD, Narine Hakobyan, PhD, Leonard A. Valentino, MD, Paul E. Monahan, MD

Blood (2007) 110 (11): 496.

https://doi.org/10.1182/blood.V110.11.496.496

Published: 2007

Journal Articles

Hemophilia B Gene Therapy Modelled in Null and Missense Mutant Mice: Kinetics of Transgenic Factor IX Expression Influences Tolerance to a Greater Extent Than Dose or Serotype of Adeno-Associated Virus (AAV) Vector.

Paul E. Monahan, MD, Tai-Ping Zhang, MD, Da-Yun Jin, Tong Gui, MD, PhD, Darrel W. Stafford, PhD

Blood (2005) 106 (11): 5526.

https://doi.org/10.1182/blood.V106.11.5526.5526

Published: 2005

NARROW

Improved Joint Health in the Phase 3 HOPE-B Trial of Etranacogene Dezaparvovec Gene Therapy for Hemophilia B over 36 Months of Follow up

Stable Factor IX Expression and Sustained Reductions in Factor IX Use 8 Years after Gene Therapy with CSL220 (Formerly AMT-060) in Adults with Hemophilia B

Adults with Severe or Moderately Severe Hemophilia B Receiving Etranacogene Dezaparvovec in the HOPE-B Phase 3 Clinical Trial Continue to Experience a Stable Increase in Mean Factor IX Activity Levels and Durable Hemostatic Protection after 24 Months’ Follow-up

BAX 335 hemophilia B gene therapy clinical trial results: potential impact of CpG sequences on gene expression

Abnormal joint and bone wound healing in hemophilia mice is improved by extending factor IX activity after hemarthrosis

Platelet-Derived Codon-Optimized Hyperfunctional FIX Gene Therapy of Hemophilia B Mice

Prophylactic efficacy of BeneFIX vs Alprolix in hemophilia B mice

Men with severe hemophilia in the United States: birth cohort analysis of a large national database

At Equal Doses FIXFc (Alprolix) Is Not Significanly Better in a Saphenous Vein Bleeding Model Than Recombinant FIX (BeneFix) Seven Days Post-Infusion

The Effect of Prophylactic Recombinant Factor IX and Glycopegylated Factor IX to Normalize Articular Wound Healing Following Hemarthrosis Examined in Hemophilia B Mice

Adeno-Associated Virus 8 (AAV8) Vector Genome Biodistribution into Body Fluids Following Single Intravenous Administration of BAX 335 Gene Therapy for Hemophilia B

Hemophilia B Mouse Lines Doubly Humanized for Human F9 and MHC Class II Genes Advance Specificity of Inhibitor Study

Continued Transmission of Parvovirus B19 in Plasma-Derived Factor Concentrates After the Implementation of B19 Nucleic Acid Plasma Minipool Screening,

Employing Factor IX Variants to Avoid Limitations Imposed by Immune Recognition of AAV Vector in Hemophilia B Gene Therapy

Intraarticular factor IX protein or gene replacement protects against development of hemophilic synovitis in the absence of circulating factor IX

Investigator-Prescribed Prophylaxis of rFIX (BeneFIX ® ) in Children <6 Years of Age: Efficacy and Safety Outcomes.

Extravascular Clotting Factor Activity within Joint Tissues Protects Against Progression of Hemophilic Arthropathy.

Hemophilia B Gene Therapy Modelled in Null and Missense Mutant Mice: Kinetics of Transgenic Factor IX Expression Influences Tolerance to a Greater Extent Than Dose or Serotype of Adeno-Associated Virus (AAV) Vector.

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American Society of Hematology

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American Society of Hematology

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