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Steven W. Pipe
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Journal Articles

Gene Therapy for Hemophilia B: Results From the Phase1/2 101HEMB 1/2 Studies

Open Access
Clinical Trials & Observations
Steven W. Pipe, M.D., Allen Poma, Anita Rajasekhar, Tamara Everington, Serap Sankoh, Jack Allen, Jason Cataldo, Eric Crombez
Journal: Blood Advances
Blood Adv bloodadvances.2024015184.
https://doi.org/10.1182/bloodadvances.2024015184
Published: 2025
Journal Articles

Completion of Phase 2b trial of etranacogene dezaparvovec gene therapy in patients with hemophilia B over 5 years

Open Access
Annette von Drygalski, M.D., Esteban Gomez, Adam Giermasz, Giancarlo Castaman, Nigel S. Key, Susan U. Lattimore, Frank W.G. Leebeek, Wolfgang A. Miesbach, Michael Recht, Paul E. Monahan, M.D., Sandra Le Quellec, M.D., Ph.D, Steven W. Pipe, M.D.
Journal: Blood Advances
Blood Adv bloodadvances.2024015291.
https://doi.org/10.1182/bloodadvances.2024015291
Published: 2025
Includes: Supplemental data
Journal Articles

Long-term safety and efficacy of fitusiran prophylaxis, and perioperative management, in people with hemophilia A or B

Open Access
Clinical Trials & Observations
Steven W. Pipe, Toshko Lissitchkov, Pencho Georgiev, Sarah Mangles, Inga Hegemann, Alice Trinchero, Pratima Chowdary, Adam Forbes, Liqi Feng, Laurel A. Menapace, Salim Kichou, Shauna Andersson, Marek Demissie, Margaret V. Ragni
Journal: Blood Advances
Blood Adv (2025) 9 (5): 1147–1158.
https://doi.org/10.1182/bloodadvances.2024013900
Published: 2025
Includes: Supplemental data
Journal Articles

Stable and durable factor IX levels in patients with hemophilia B over 3 years after etranacogene dezaparvovec gene therapy

Free
Clinical Trials & Observations
Annette von Drygalski, Esteban Gomez, Adam Giermasz, Giancarlo Castaman, Nigel S. Key, Susan U. Lattimore, Frank W. G. Leebeek, Wolfgang A. Miesbach, Michael Recht, Robert Gut, Ricardo Dolmetsch, Paul E. Monahan, Sandra Le Quellec, Steven W. Pipe
Journal: Blood Advances
Blood Adv (2023) 7 (19): 5671–5679.
https://doi.org/10.1182/bloodadvances.2022008886
Published: 2023
Includes: Supplemental data
Journal Articles

Molecular evaluation and vector integration analysis of HCC complicating AAV gene therapy for hemophilia B

Free
Clinical Trials & Observations
Manfred Schmidt, Graham R. Foster, Michiel Coppens, Hauke Thomsen, Ricardo Dolmetsch, Liesbeth Heijink, Paul E. Monahan, Steven W. Pipe
Journal: Blood Advances
Blood Adv (2023) 7 (17): 4966–4969.
https://doi.org/10.1182/bloodadvances.2023009876
Published: 2023
Includes: Supplemental data
Journal Articles

Surgical outcomes in people with hemophilia A taking emicizumab prophylaxis: experience from the HAVEN 1-4 studies

Free
Rebecca Kruse-Jarres, Flora Peyvandi, Johannes Oldenburg, Tiffany Chang, Sammy Chebon, Michelle Y. Doral, Stacy E. Croteau, Thierry Lambert, Christine L. Kempton, Steven W. Pipe, Richard H. Ko, Benjamin Trzaskoma, Christophe Dhalluin, Nives Selak Bienz, Markus Niggli, Michaela Lehle, Ido Paz-Priel, Guy Young, Víctor Jiménez-Yuste
Journal: Blood Advances
Blood Adv (2022) 6 (24): 6140–6150.
https://doi.org/10.1182/bloodadvances.2022007458
Published: 2022
Includes: Supplemental data
Journal Articles

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

Free
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

Efficacy of emicizumab in a pediatric patient with type 3 von Willebrand disease and alloantibodies

Free
Angela C. Weyand, Veronica H. Flood, Jordan A. Shavit, Steven W. Pipe
Journal: Blood Advances
Blood Adv (2019) 3 (18): 2748–2750.
https://doi.org/10.1182/bloodadvances.2019000656
Published: 2019