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Journal Articles

Long-Term Bleeding Protection, Sustained FIX Activity, Reduction of FIX Consumption and Safety of Hemophilia B Gene Therapy: Results from the HOPE-B Trial 3 Years after Administration of a Single Dose of Etranacogene Dezaparvovec in Adult Patients with Severe or Moderately Severe Hemophilia B

Blood (2023) 142 (Supplement 1): 1055.

https://doi.org/10.1182/blood-2023-187624

Published: 2023

Journal Articles

Low Rebleed Incidence with Eptacog Beta Treatment through 24 and 48 Hours in Children (<12 years) with Hemophilia a or B with Inhibitors

Blood (2023) 142 (Supplement 1): 5480.

https://doi.org/10.1182/blood-2023-181299

Published: 2023

Journal Articles

HIV Comorbid Infection and Liver-Directed AAV-Based Gene Therapy in Adults with Severe and Moderately Severe Hemophilia B: Efficacy and Safety Results from Phase 2b and the Pivotal Phase 3 HOPE-B Trials 3 Years after Administration of a Single Dose of Etranacogene Dezaparvovec

Steven Pipe, Esteban Gomez, Cedric R. Hermans, Adam Giermasz, Peter Kampmann, Richard Lemons, Nicholas Galante, Sandra LeQuellec, Paul Monahan

Blood (2023) 142 (Supplement 1): 2256.

https://doi.org/10.1182/blood-2023-187059

Published: 2023

Journal Articles

Emicizumab Prophylaxis in Infants with Severe Hemophilia A without Factor VIII Inhibitors: Results from the Primary Analysis of the HAVEN 7 Study

Steven Pipe, Peter Collins, Christophe Dhalluin, Gili Kenet, Christophe Schmitt, Muriel Buri, Víctor Jiménez-Yuste, Flora Peyvandi, Guy Young, Johannes Oldenburg, Maria Elisa Mancuso, Kaan Kavakli, Anna Kiialainen, Tiffany Chang, Michaela Lehle, Markus Niggli, Karin Fijnvandraat

Blood (2023) 142 (Supplement 1): 505.

https://doi.org/10.1182/blood-2023-177963

Published: 2023

Journal Articles

Pharmacodynamic Biomarkers in Infants with Hemophilia A Receiving Emicizumab in HAVEN 7

Anna Kiialainen, Steven Pipe, Karin Fijnvandraat, Gili Kenet, Christophe Schmitt, Muriel Buri, Víctor Jiménez-Yuste, Johannes Oldenburg, Maria Elisa Mancuso, Christophe Dhalluin, Michaela Lehle, Peter Collins

Blood (2023) 142 (Supplement 1): 1238.

https://doi.org/10.1182/blood-2023-173015

Published: 2023

Journal Articles

Fitusiran, an RNAi Therapeutic Targeting Antithrombin to Restore Hemostatic Balance in Patients with Hemophilia a or B with or without Inhibitors: Management of Acute Bleeding Events

Steven Pipe, MD, Margaret V. Ragni, MD MPH, Claude Négrier, MD PhD, Qifeng Yu, PhD, Naghmana Bajwa, MD, John Caminis, MD, Baisong Mei, MD PhD, Shauna R. Andersson, MD PhD

Blood (2019) 134 (Supplement_1): 1138.

https://doi.org/10.1182/blood-2019-124108

Published: 2019

Journal Articles

First-in-human Gene Therapy Study of AAVhu37 Capsid Vector Technology in Severe Hemophilia A

Steven Pipe, MD, Michael Becka, Elke Detering, Konstantina Vanevski, Toshko Lissitchkov

Blood (2019) 134 (Supplement_1): 4630.

https://doi.org/10.1182/blood-2019-125764

Published: 2019

Journal Articles

One Year Data from a Phase 2b Trial of AMT-061 (AAV5-Padua hFIX variant), an Enhanced Vector for Gene Transfer in Adults with Severe or Moderate-Severe Hemophilia B

Steven Pipe, MD, Adam Giermasz, MD PhD, Giancarlo Castaman, MD, Nigel S. Key, MBChB, FRCP, Susan U Lattimore, Frank Leebeek, MD PhD, Wolfgang Miesbach, MD PhD, Michael Recht, MD PhD, Esteban Gomez, MD, Alison Long, Robert Gut, Annette von Drygalski, MD PharmD

Blood (2019) 134 (Supplement_1): 3348.

https://doi.org/10.1182/blood-2019-128765

Published: 2019

Journal Articles

Real-World Data of Immune Tolerance Induction Using rFVIIIFc in Subjects With Severe Hemophilia A With Inhibitors at High Risk for ITI Failure

Manuel Carcao, Amy Shapiro, Nina Hwang, Steven Pipe, Sanjay Ahuja, Kenneth Lieuw, MD PhD, Janice Staber, Haowei Linda Sun, Hilda Ding, Michael Wang, Macgregor Steele, Elisa Tsao, Jing Feng, Zahra Al-Khateeb, Jennifer Dumont, Nisha Jain

Blood (2018) 132 (Supplement 1): 2500.

https://doi.org/10.1182/blood-2018-99-110467

Published: 2018

Journal Articles

Preference for Emicizumab over Prior Factor Treatments: Results from the HAVEN 3 and HAVEN 4 Studies

Victor Jimenez-Yuste, Midori Shima, MD PhD, Ido Paz-Priel, Aric Parnes, Michaela Lehle, Adam Giermasz, Avrita Campinha-Bacote, Markus Niggli, Jerzy Windyga, Sammy Chebon, Peter Trask, Johnny Mahlangu, Steven Pipe, Johannes Oldenburg

Blood (2018) 132 (Supplement 1): 1187.

https://doi.org/10.1182/blood-2018-99-118132

Published: 2018

Journal Articles

The Irish Personalized Approach to the Treatment of Haemophilia (iPATH) - Determinants of Inter-Individual Variation in FVIII Pharmacokinetics

Michelle Lavin, MB, PhD FRCPath, Niamh Larkin, Anjali Patel, Evelyn Singleton, Mary Byrne, Kevin Ryan, MB, FRCPath, Niamh M O'Connell, MB, PhD FRCPath, Birgit M Reipert, Steven Pipe, Peter L Turecek, PhD, James S O'Donnell, MD PhD

Blood (2018) 132 (Supplement 1): 1190.

https://doi.org/10.1182/blood-2018-99-112850

Published: 2018

Journal Articles

101HEMB01 Is a Phase 1/2 Open-Label, Single Ascending Dose-Finding Trial of DTX101 (AAVrh10FIX) in Patients with Moderate/Severe Hemophilia B That Demonstrated Meaningful but Transient Expression of Human Factor IX (hFIX)

Steven Pipe, MD, Kimo Stine, MD, Anita Rajasekhar, MD, Tamara Everington, MD, Allen Poma, MD, Eric Crombez, MD, Charles RM Hay, MD

Blood (2017) 130 (Supplement 1): 3331.

https://doi.org/10.1182/blood.V130.Suppl_1.3331.3331

Published: 2017

Journal Articles

Safety and Efficacy of Recombinant Factor VIIa (rFVIIa) in Congenital Hemophilia with Inhibitors (CHwI) in the Home Treatment Setting: Systematic Review of Clinical Studies and Registries

Guy Young, MD, Miguel Escobar, Steven Pipe, MD, David L. Cooper, MD MBA

Blood (2015) 126 (23): 2302.

https://doi.org/10.1182/blood.V126.23.2302.2302

Published: 2015

Journal Articles

Improving Hematologist/Oncologist Knowledge and Confidence in Managing Hemophilia through Online Educational Interventions

Emily S Van Laar, MS, Charlotte Warren, John Maeglin, Mila Kostic, Michael Schoen, PhD, Neil Frick, MS, Steven Pipe, MD

Blood (2015) 126 (23): 5593.

https://doi.org/10.1182/blood.V126.23.5593.5593

Published: 2015

Journal Articles

Thrombin-Stimulated Platelets Have Functional Binding Sites For Factor VIIIa That Are Distinct From Phosphatidylserine

Jialan Shi, PhD, Valerie A Novakovic, M.S., Steven Pipe, Shannon Meeks, M.D., John (Pete) S. Lollar, MD, Stephanie A. Smith, DVM, MS, James H. Morrissey, PhD, Gary E. Gilbert, M.D.

Blood (2013) 122 (21): 3582.

https://doi.org/10.1182/blood.V122.21.3582.3582

Published: 2013

Journal Articles

Elucidation of the Roles of Individual Asparagine-Linked Glycans Outside of the B Domain on Factor VIII Secretion

Sundar Rajan Selvaraj, PhD, Hongzhi Miao, MS, Steven Pipe, MD

Blood (2011) 118 (21): 2238.

https://doi.org/10.1182/blood.V118.21.2238.2238

Published: 2011

Journal Articles

Conservative Mutations in the Membrane-Binding Motif of Factor V C2 Domain to Residues of Pseudonaja Textilis Venom Factor V Confer Phosphatidylserine-Independent Activity

Valerie A Novakovic, M.S., Hongzhi Miao, Steven Pipe, MD, Gary E. Gilbert, M.D.

Blood (2011) 118 (21): 2243.

https://doi.org/10.1182/blood.V118.21.2243.2243

Published: 2011

Journal Articles

Endoplasmic Reticulum to Mitochondria Ca 2+ Signaling Inhibits Factor VIII Secretion and Mediates Oxidative Stress and Apoptosis Upon Factor VIII Expression

Jyoti Malhotra, Mon Xiong, Hongzhi Miao, Steven Pipe, Randal J. Kaufman, PhD

Blood (2010) 116 (21): 2212.

https://doi.org/10.1182/blood.V116.21.2212.2212

Published: 2010

Journal Articles

Hemostasis and Thrombosis Centers Pilot Sites Registry: Thrombophilia Evaluation in Children.

Courtney Thornburg, MD, MS, Michele Beckman, PhD, Lisa Michaels, MD, Marilyn Manco-Johnson, MD, Nicole Dowling, PhD, Steven Pipe, MD, Roshni Kulkarni, MD

Blood (2006) 108 (11): 3298.

https://doi.org/10.1182/blood.V108.11.3298.3298

Published: 2006

Journal Articles

An Essential Role of the Factor VIII Light Chain in Facilitating Heavy Chain Secretion.

Lingxia Chen, PhD, Juan Li, M.D, Hui Lu, Haiyan Jiang, Ph.D, Rita Sarkar, Ph.D, Jinhui Wang, Ph.D, Valder Arruda, M.D, Glenn Pierce, M.D, Steven Pipe, M.D, Paul Liu, Ph.D, Xiao Xiao, Ph.D, Rodney Camire, Ph.D, Weidong Xiao, Ph.D

Blood (2006) 108 (11): 4034.

https://doi.org/10.1182/blood.V108.11.4034.4034

Published: 2006

NARROW

Long-Term Bleeding Protection, Sustained FIX Activity, Reduction of FIX Consumption and Safety of Hemophilia B Gene Therapy: Results from the HOPE-B Trial 3 Years after Administration of a Single Dose of Etranacogene Dezaparvovec in Adult Patients with Severe or Moderately Severe Hemophilia B

Low Rebleed Incidence with Eptacog Beta Treatment through 24 and 48 Hours in Children (<12 years) with Hemophilia a or B with Inhibitors

HIV Comorbid Infection and Liver-Directed AAV-Based Gene Therapy in Adults with Severe and Moderately Severe Hemophilia B: Efficacy and Safety Results from Phase 2b and the Pivotal Phase 3 HOPE-B Trials 3 Years after Administration of a Single Dose of Etranacogene Dezaparvovec

Emicizumab Prophylaxis in Infants with Severe Hemophilia A without Factor VIII Inhibitors: Results from the Primary Analysis of the HAVEN 7 Study

Pharmacodynamic Biomarkers in Infants with Hemophilia A Receiving Emicizumab in HAVEN 7

Fitusiran, an RNAi Therapeutic Targeting Antithrombin to Restore Hemostatic Balance in Patients with Hemophilia a or B with or without Inhibitors: Management of Acute Bleeding Events

First-in-human Gene Therapy Study of AAVhu37 Capsid Vector Technology in Severe Hemophilia A

One Year Data from a Phase 2b Trial of AMT-061 (AAV5-Padua hFIX variant), an Enhanced Vector for Gene Transfer in Adults with Severe or Moderate-Severe Hemophilia B

Real-World Data of Immune Tolerance Induction Using rFVIIIFc in Subjects With Severe Hemophilia A With Inhibitors at High Risk for ITI Failure

Preference for Emicizumab over Prior Factor Treatments: Results from the HAVEN 3 and HAVEN 4 Studies

The Irish Personalized Approach to the Treatment of Haemophilia (iPATH) - Determinants of Inter-Individual Variation in FVIII Pharmacokinetics

101HEMB01 Is a Phase 1/2 Open-Label, Single Ascending Dose-Finding Trial of DTX101 (AAVrh10FIX) in Patients with Moderate/Severe Hemophilia B That Demonstrated Meaningful but Transient Expression of Human Factor IX (hFIX)

Safety and Efficacy of Recombinant Factor VIIa (rFVIIa) in Congenital Hemophilia with Inhibitors (CHwI) in the Home Treatment Setting: Systematic Review of Clinical Studies and Registries

Improving Hematologist/Oncologist Knowledge and Confidence in Managing Hemophilia through Online Educational Interventions

Thrombin-Stimulated Platelets Have Functional Binding Sites For Factor VIIIa That Are Distinct From Phosphatidylserine

Elucidation of the Roles of Individual Asparagine-Linked Glycans Outside of the B Domain on Factor VIII Secretion

Conservative Mutations in the Membrane-Binding Motif of Factor V C2 Domain to Residues of Pseudonaja Textilis Venom Factor V Confer Phosphatidylserine-Independent Activity

Endoplasmic Reticulum to Mitochondria Ca 2+ Signaling Inhibits Factor VIII Secretion and Mediates Oxidative Stress and Apoptosis Upon Factor VIII Expression

Hemostasis and Thrombosis Centers Pilot Sites Registry: Thrombophilia Evaluation in Children.

An Essential Role of the Factor VIII Light Chain in Facilitating Heavy Chain Secretion.

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American Society of Hematology

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American Society of Hematology

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