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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

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

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

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

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

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

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

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

Prevention of Early Arteriovenous Fistula Failure Due to Thromosis: Experience with Primary Thromboprophylaxis.

Anjali A. Sharathkumar, MD, Jin-Jar Lin, MD, PhD, Ronald Hirschl, MD, Steven Pipe, MD

Blood (2006) 108 (11): 885.

https://doi.org/10.1182/blood.V108.11.885.885

Published: 2006

NARROW

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

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

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

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)

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

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

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

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

Prevention of Early Arteriovenous Fistula Failure Due to Thromosis: Experience with Primary Thromboprophylaxis.

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American Society of Hematology

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ASH Publications

American Society of Hematology

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