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All Publications

Displaying 14 results.
    • Journal article
    Eptacog beta efficacy and safety in the treatment and control of bleeding in paediatric subjects (<12 years) with haemophilia A or B with inhibitors.
    Pipe, Steven W Hermans, Cédric[UCL] Chitlur, Meera Carcao, Manuel Castaman, Giancarlo Davis, Joanna A Ducore, Jonathan Dunn, Amy L Escobar, Miguel Journeycake, Janna Khan, Osman Mahlangu, Johnny Meeks, Shannon L Mitha, Ismail Haroon Négrier, Claude Nowak-Göttl, Ulrike Recht, Michael Chrisentery-Singleton, Tammuella Stasyshyn, Oleksandra Vilchevska, Kateryna V Martinez, Laura Villarreal Wang, Michael Windyga, Jerzy Young, Guy Alexander, W Allan Bonzo, Daniel Macie, Christopher Mitchell, Ian S Sauty, Evelyne Wilkinson, Thomas A Shapiro, Amy D (2022) Haemophilia — Vol. 28, no. 4, p. 548-556 (2022)
    • Journal article
    Vaccination against COVID-19: Rationale, modalities and precautions for patients with haemophilia and other inherited bleeding disorders.
    Kaczmarek, Radoslaw El Ekiaby, Magdy Hart, Daniel P Hermans, Cédric[UCL] Makris, Mike Noone, Declan O'Mahony, Brian Page, David Peyvandi, Flora Pipe, Steven W Sannié, Thomas Schlenkrich, Uwe Skinner, Mark W Srivastava, Alok Bok, Amanda Pierce, Glenn F World Federation of Hemophilia (WFH) European Association for Haemophilia, Allied Disorders (EAHAD) European Haemophilia Consortium (EHC) U.S. National Hemophilia Foundation (NHF) (2021) Haemophilia — Vol. 27, no. 4, p. 515-518 (2021)
    • Journal article
    Management of COVID-19-associated coagulopathy in persons with haemophilia.
    Pipe, Steven W Kaczmarek, Radoslaw Srivastava, Alok Pierce, Glenn F Makris, Mike Hermans, Cédric[UCL] Interim Guidance Coagulation Products Safety, Supply and Access (CPSSA) Committee of the World Federation of Hemophilia (2021) Haemophilia — Vol. 27, no.1, p. 41-48 (2021)
    • Journal article
    Evolution of haemophilia integrated care in the era of gene therapy: Treatment centre's readiness in United States and EU.
    Miesbach, Wolfgang Pasi, K John Pipe, Steven W Hermans, Cédric[UCL] O'Mahony, Brian Guelcher, Christine Steiner, Bruno Skinner, Mark W (2021) Haemophilia — Vol. 27, no. 4, p. 511-514 (2021)
    • Journal article
    Etranacogene dezaparvovec gene therapy for haemophilia B (HOPE-B): 24-month post-hoc efficacy and safety data from a single-arm, multicentre, phase 3 trial.
    Coppens, Michiel Pipe, Steven W Miesbach, Wolfgang Astermark, Jan Recht, Michael van der Valk, Paul Ewenstein, Bruce Pinachyan, Karen Galante, Nicholas Le Quellec, Sandra Monahan, Paul E Leebeek, Frank W G HOPE-B Investigators Hermans, Cédric[UCL] (2024) The Lancet. Haematology — Vol. 11, no.4, p. e265-e275 (2024)
    • Journal article
    Efficacy, safety, and pharmacokinetics of emicizumab prophylaxis given every 4 weeks in people with haemophilia A (HAVEN 4): a multicentre, open-label, non-randomised phase 3 study.
    Pipe, Steven W Shima, Midori Lehle, Michaela Shapiro, Amy Chebon, Sammy Fukutake, Katsuyuki Key, Nigel S Portron, Agnès Schmitt, Christophe Podolak-Dawidziak, Maria Selak Bienz, Nives Hermans, Cédric[UCL] Campinha-Bacote, Avrita Kiialainen, Anna Peerlinck, Kathelijne Levy, Gallia G Jiménez-Yuste, Victor (2019) The Lancet. Haematology — Vol. 6, no.6, p. e295-e305 (2019)
    • Journal article
    Long-term outcomes with emicizumab in hemophilia A without inhibitors: results from the HAVEN 3 and 4 studies.
    Mahlangu, Johnny Jiménez-Yuste, Víctor Ventriglia, Giuliana Niggli, Markus Barlera, Simona Hermans, Cédric[UCL] Lehle, Michaela Chowdary, Pratima Jew, Lyle Windyga, Jerzy Frenzel, Laurent Schmitt, Christophe Castaman, Giancarlo Pipe, Steven W (2024) Research and practice in thrombosis and haemostasis — Vol. 8, no.2, p. 102364 [1-12] (2024)
    • Journal article
    Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B.
    Pipe, Steven W Leebeek, Frank W G Recht, Michael Key, Nigel S Castaman, Giancarlo Miesbach, Wolfgang Lattimore, Susan Peerlinck, Kathelijne Van der Valk, Paul Coppens, Michiel Kampmann, Peter Meijer, Karina O'Connell, Niamh Pasi, K John Hart, Daniel P Kazmi, Rashid Astermark, Jan Hermans, Cédric[UCL] Klamroth, Robert Lemons, Richard Visweshwar, Nathan von Drygalski, Annette Young, Guy Crary, Shelley E Escobar, Miguel Gomez, Esteban Kruse-Jarres, Rebecca Quon, Doris V Symington, Emily Wang, Michael Wheeler, Allison P Gut, Robert Liu, Ying P Dolmetsch, Ricardo E Cooper, David L Li, Yanyan Goldstein, Brahm Monahan, Paul E (2023) The New England journal of medicine — Vol. 388, no.8, p. 706-718 (2023)
    • Journal article
    Two-Year Outcomes of Valoctocogene Roxaparvovec Therapy for Hemophilia A.
    Mahlangu, Johnny Kaczmarek, Radoslaw von Drygalski, Annette Shapiro, Susan Chou, Sheng-Chieh Ozelo, Margareth C Kenet, Gili Peyvandi, Flora Wang, Michael Madan, Bella Key, Nigel S Laffan, Michael Dunn, Amy L Mason, Jane Quon, Doris V Symington, Emily Leavitt, Andrew D Oldenburg, Johannes Chambost, Hervé Reding, Mark T Jayaram, Kala Yu, Hua Mahajan, Reena Chavele, Konstantia-Maria Reddy, Divya B Henshaw, Joshua Robinson, Tara M Wong, Wing Yen Pipe, Steven W GENEr8-1 Trial Group Hermans, Cédric[UCL] (2023) The New England journal of medicine — Vol. 388, no.8, p. 694-705 (2023)
    • Journal article
    Extending health equity to people with moderate and mild hemophilia A: revisiting the HAVEN 6 trial.
    Hermans, Cédric[UCL] Coppens, Michiel Ventriglia, Giuliana Ling, Gavin Lehle, Michaela Pipe, Steven W (2025) Research and practice in thrombosis and haemostasis — Vol. 9, no.1, p. 102648 [1-6] (2025)
    • Journal article
    Recombinant factor VIIa: new insights into the mechanism of action through product innovation.
    Escobar, Miguel A Hoffman, Maureane Castaman, Giancarlo Hermans, Cédric[UCL] Mahlangu, Johnny Oldenburg, Johannes Percy, Charles L Reding, Mark T Shapiro, Amy D Pipe, Steven W (2025) Research and practice in thrombosis and haemostasis — Vol. 9, no.1, p. 102670 [1-14] (2025)
    • Journal article
    Operationalising a Haemophilia Gene Editing Lexicon for Practical Use.
    McKeown, William Hermans, Cédric[UCL] Unzu, Carmen Kay, Mark A Peyvandi, Flora Smith, Penni Miesbach, Wolfgang Pierce, Glenn F Khair, Kate Valentino, Leonard A Pipe, Steven W Pillai, Monisha Jones, Micheala Delwart, Virginie Sindhurakar, Anil Gutstein, David E Kessler, Craig M (2025) Haemophilia — , p. [1-7] (2025)
    • Journal article
    Accurate evaluation of factor VIII activity of efanesoctocog alfa in the presence of emicizumab.
    Nougier, Christophe Pipe, Steven W Pabinger, Ingrid Pouplard, Claire Schutgens, Roger E G Hermans, Cédric[UCL] Young, Guy Sexton, Edison Quehenberger, Peter Guery, Eve-Anne Huisman, Albert Van Dievoet, Marie-Astrid Marquez, Elizabeth Dargaud, Yesim (2025) Journal of thrombosis and haemostasis — , p. n (2025)
    • Journal article
    A novel gene editing lexicon strategy for the haemophilia community: Research plan for development and preliminary results.
    Hermans, Cédric[UCL] Valentino, Leonard A Thornburg, Courtney D Unzu, Carmen Kay, Mark A Peyvandi, Flora Smith, Penni Miesbach, Wolfgang McKeown, William Pierce, Glenn F Khair, Kate Pipe, Steven W Starcevic, Katarina Pillai, Monisha Jones, Micheala Chiao, Megan Antonino, Ilia Kessler, Craig (2024) Haemophilia — Vol. 30, no.6, p. 1272-1280 (2024)