Publications by authors named "Steven Pipe"

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Final Analysis of a Study of Etranacogene Dezaparvovec for Hemophilia B.
Steven W Pipe, Wolfgang Miesbach, Michael Recht, Frank W G Leebeek, Nigel S Key

N Engl J Med· December 2025

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The WFH Guidelines for the Management of Haemophilia: AAV Gene Therapy, 2025.
Glenn F Pierce, Margareth C Ozelo, Johnny Mahlangu, Amy L Dunn, Marlene Beijlevelt, Steven Pipe

Haemophilia· October 2025

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A comprehensive care pathway of gene therapy for hemophilia based on current guideline documents and summary of product characteristics: communication from the ISTH SSC working group on gene therapy.
Caroline M A Mussert, Wolfgang Miesbach, Pratima Chowdary, David Lillicrap, Johnny Mahlangu, Steven W Pipe

J Thromb Haemost· October 2025

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Natural history of preexisting AAV5 antibodies in adults with hemophilia B during the lead-in of the etranacogene dezaparvovec phase 3 study.
Robert Klamroth, Michael Recht, Nigel S Key, Wolfgang Miesbach, Steven W Pipe

Mol Ther Methods Clin Dev· September 2025

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Treatment of severe bleeds with eptacog beta in hemophilia A or B with inhibitors: a post hoc analysis of the PERSEPT 1 and 2 trials.
Guy Young, Johnny Mahlangu, Lisa N Boggio, Manuel Carcao, Yesim Dargaud, Steven W Pipe

Blood Vessel Thromb Hemost· August 2025

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Characterization of the clinical and laboratory phenotype of hemophilia carriers and trends of utilization of hemostatic therapies: analysis of the American Thrombosis and Hemostasis Network dataset.
Neeraja Swaminathan, Alison Currie, Jianzhong Hu, Roshni Kulkarni, Steven Pipe

Res Pract Thromb Haemost· August 2025

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Corticosteroid use to mitigate transaminitis-associated decline in FVIII levels following valoctocogene roxaparvovec gene therapy: clinical practice guidance.
Barbara A Konkle, Flora Peyvandi, Graham R Foster, Cedric Hermans, Vincenzo La Mura, Steven Pipe

J Thromb Haemost· July 2025

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Completion of phase 2b trial of etranacogene dezaparvovec gene therapy in patients with hemophilia B over 5 years.
Annette von Drygalski, Esteban Gomez, Adam Giermasz, Giancarlo Castaman, Nigel S Key, Steven W Pipe

Blood Adv· July 2025

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Pain Reduction Following Eptacog Beta Treatment of Bleeding Episodes in Adolescents and Adults With Haemophilia A or B Complicated by Inhibitors.
Tyler W Buckner, Craig Kessler, Giancarlo Castaman, Cédric Hermans, Victor Jiménez-Yuste, Steven W Pipe

Haemophilia· July 2025

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Safety and efficacy of a fitusiran antithrombin-based dose regimen in people with hemophilia A or B: the ATLAS-OLE study.
Guy Young, Kaan Kavakli, Robert Klamroth, Tadashi Matsushita, Flora Peyvandi, Steven W Pipe

Blood· June 2025

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Gene therapy for hemophilia B: results from the phase 1/2 101HEMB01/02 studies.
Steven Pipe, Allen Poma, Anita Rajasekhar, Tamara Everington, Serap Sankoh

Blood Adv· June 2025

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Accurate evaluation of factor VIII activity of efanesoctocog alfa in the presence of emicizumab.
Christophe Nougier, Steven W Pipe, Ingrid Pabinger, Claire Pouplard, Roger E G Schutgens

J Thromb Haemost· May 2025

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Global Comparative Antithrombin Field Study: Impact of Laboratory Assay Variability on the Assessment of Antithrombin Activity Measurement at Fitusiran Clinical Decision-Making Points.
Ekta Seth Chhabra, Ali Sadeghi-Khomami, Mingjie Liu, Guy Young, Steven W Pipe

Haemophilia· May 2025

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Gene-based therapies for hemophilia.
Melissa F Glasner, Steven Pipe, Wolfgang Miesbach

Res Pract Thromb Haemost· May 2025

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Guidelines for management of hemophilia-why, what, and how?
Alok Srivastava, Johnny Mahlangu, Steven W Pipe

Res Pract Thromb Haemost· May 2025

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Activated prothrombin complex concentrate in patients receiving emicizumab prophylaxis: from evidence to clinical practice.
Robert F Sidonio, Guy Young, Carmen Escuriola Ettingshausen, Johnny Mahlangu, Margareth C Ozelo, Steven W Pipe

Res Pract Thromb Haemost· May 2025

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Long-term safety and efficacy of fitusiran prophylaxis, and perioperative management, in people with hemophilia A or B.
Steven W Pipe, Toshko Lissitchkov, Pencho Georgiev, Sarah Mangles, Inga Hegemann

Blood Adv· March 2025

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Operationalising a Haemophilia Gene Editing Lexicon for Practical Use.
William McKeown, Cedric Hermans, Carmen Unzu, Mark A Kay, Flora Peyvandi, Steven W Pipe

Haemophilia· March 2025

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Haemophilia.
Pratima Chowdary, Manuel Carcao, Gili Kenet, Steven W Pipe

Lancet· March 2025

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Development of a novel gene editing lexicon for hemophilia: methodology and results.
Craig M Kessler, Leonard A Valentino, Courtney D Thornburg, Carmen Unzu, Mark A Kay, Steven W Pipe

Res Pract Thromb Haemost· February 2025

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Extending health equity to people with moderate and mild hemophilia A: revisiting the HAVEN 6 trial.
Cedric Hermans, Michiel Coppens, Giuliana Ventriglia, Gavin Ling, Michaela Lehle, Steven W Pipe

Res Pract Thromb Haemost· January 2025

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Adeno-associated virus-based gene therapy for hemophilia-addressing the gaps.
Wolfgang Miesbach, Paul Batty, Pratima Chowdary, Sylvia Fong, Radoslaw Kaczmarek, Steven W Pipe

Res Pract Thromb Haemost· January 2025

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Recombinant factor VIIa: new insights into the mechanism of action through product innovation.
Miguel A Escobar, Maureane Hoffman, Giancarlo Castaman, Cedric Hermans, Johnny Mahlangu, Steven W Pipe

Res Pract Thromb Haemost· January 2025

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Outcomes and management of invasive procedures in participants with hemophilia A post gene therapy: a post hoc analysis of the GENEr8-1 phase III trial.
Doris V Quon, Jiaan-Der Wang, Michael Wang, Dominic Pepperell, Young-Shil Park, Steven W Pipe

Ther Adv Hematol· December 2024

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Efficacy, safety, and quality of life 4 years after valoctocogene roxaparvovec gene transfer for severe hemophilia A in the phase 3 GENEr8-1 trial.
Andrew D Leavitt, Johnny Mahlangu, Priyanka Raheja, Emily Symington, Doris V Quon, Steven W Pipe

Res Pract Thromb Haemost· November 2024

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