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Journal Articles

First Data from the Phase 3 HOPE-B Gene Therapy Trial: Efficacy and Safety of Etranacogene Dezaparvovec (AAV5-Padua hFIX variant; AMT-061) in Adults with Severe or Moderate-Severe Hemophilia B Treated Irrespective of Pre-Existing Anti-Capsid Neutralizing Antibodies

Steven W. Pipe, MD, Michael Recht, MD PhD, Nigel S. Key, MD, Frank W.G. Leebeek, MD PhD, Giancarlo Castaman, MD, Susan U. Lattimore, R.N., Paul Van Der Valk, Kathelijne Peerlinck, MD PhD, Michiel Coppens, MD, Niamh O'Connell, MD PhD, John Pasi, MB, ChB, PhD FRCP, FRCPath, FRCPCH, Peter Kampmann, MD, Karina Meijer, MD PhD, Annette von Drygalski, MD Pharm D, Guy Young, MD, Cedric Hermans, MDMRCP,PhD, Jan Astermark, MD PhD, Robert Klamroth, MD PhD, Richard S. Lemons, MD, Nathan Visweshwar, MD, Shelley Crary, MD MS, Rashid Kazmi, MBBS, Emily Symington, Miguel A. Escobar, MD, Esteban Gomez, MD, Rebecca Kruse-Jarres, MD, Adam Kotowski, MD, Doris Quon, MD PhD, Michael Wang, MD, Allison P. Wheeler, MD, Eileen K Sawyer, PhD, Stephanie Verweij, BSc, Valerie Colletta, MSc, Naghmana Bajma, MD, Robert Gut, MD PhD, Wolfgang A. Miesbach, MD PhD

Blood (2020) 136 (Supplement_2): LBA-6.

https://doi.org/10.1182/blood-2020-143560

Published: 2020

Journal Articles

Lymphatic Remodeling Associated with Hemophilic Joint Bleeding in FVIII-Deficient Mice

Esther J Cooke, PhD, Chanond A Nasamran, Kathleen Fisch, PhD, Annette von Drygalski, MD PharmD, RMSK

Blood (2020) 136 (Supplement 1): 20.

https://doi.org/10.1182/blood-2020-143369

Published: 2020

Journal Articles

Validity of Quantitative Measurements By the Joint Tissue Examination and Damage Exam (JADE) with Musculoskeletal Ultrasound for the Longitudinal Assessment of Hemophilic Arthropathy

Akram Mesleh Shayeb, MD, Richard Barnes, PhD MPH, Nicolas Gallastegui Crestani, MD, Cris Hanacek, BS, Peter Aguero, PT, DPT, Andres Flores, MS, ATC, CES, Rebecca Kruse-Jarres, MD, Bruno Steiner, PT, DPT, LMT, RMSK, Doris Quon, MD PhD, Cindy Bailey, DPT, OCS, SCS, ATC, Annette von Drygalski, MD PharmD, RMSK

Blood (2020) 136 (Supplement 1): 20–21.

https://doi.org/10.1182/blood-2020-143133

Published: 2020

Journal Articles

Incidence of Venous Thromboembolic Events in Hospitalized COVID-19 Patients: A Systematic Review

Nicolas Galllastegui Crestani, MD, Jenny Y Zhou, MD, Annette von Drygalski, MD PharmD, RMSK, Timothy A Morris, MD

Blood (2020) 136 (Supplement 1): 42–43.

https://doi.org/10.1182/blood-2020-143021

Published: 2020

Journal Articles

Traumatic Bleeding in Mice Is Amplified By Iron Deficiency Anemia and Can be Mitigated By Tranexamic Acid Prophylaxis

Bilgimol Chumappumkal Joseph, PhD, Annette von Drygalski, MD PharmD, RMSK

Blood (2020) 136 (Supplement 1): 35.

https://doi.org/10.1182/blood-2020-137052

Published: 2020

Journal Articles

Risk of Hypertension in Hemophilia Patients with a History of Inhibitors

Anna Ter-Zakarian, Richard Barnes, PhD MPH, Rebecca Kruse-Jarres, MD MPH, Doris Quon, MD PhD, Shannon Jackson, MD FRCPC, Linda Sun, MD, Annette von Drygalski, MD PharmD, RMSK

Blood (2020) 136 (Supplement 1): 30–31.

https://doi.org/10.1182/blood-2020-136133

Published: 2020

Journal Articles

Mechanisms of Iron Clearance from the Joint in FVIII-Deficient Mice after Induced Hemarthrosis

Esther J Cooke, PhD, Chanond A Nasamran, Kathleen M Fisch, Annette von Drygalski, MD PharmD

Blood (2019) 134 (Supplement_1): 157.

https://doi.org/10.1182/blood-2019-131576

Published: 2019

Journal Articles

Prevention of Bleeding-Induced Vascular Abnormalities in the Hemophilic Mouse Joint By Increasing TAFI Levels or Inhibiting uPA Activity

Tine Wyseure, PhD, Niels Behrendt, Annette von Drygalski, MD PharmD, Laurent O Mosnier

Blood (2019) 134 (Supplement_1): 158.

https://doi.org/10.1182/blood-2019-128723

Published: 2019

Journal Articles

Super factor Va, an Engineered Factor V Molecule, Effectively Reduces Bleeding and Acute Coagulopathy in Mouse Models of Trauma and Shock

Bilgimol Chumappumkal Joseph, PhD, Laurent O Mosnier, Annette von Drygalski, MD PharmD

Blood (2019) 134 (Supplement_1): 1101.

https://doi.org/10.1182/blood-2019-124433

Published: 2019

Journal Articles

Quantitative Measurements of Hemophilic Joint Tissues By Point-of-Care Musculoskeletal Ultrasound Are Associated with Clinical and Functional Joint Outcome Parameters

Akram Mesleh Shayeb, MD, Richard Barnes, PhD MPH, Cris Hanacek, BS, Andres Flores, MS, ATC, CES, Rebecca Kruse-Jarres, MDMPH, Bruno Steiner, PT, DPT, LMT, RMSK, Doris Quon, MD PhD, Cindy Bailey, DPT, OCS, SCS, ATC, Annette von Drygalski, MD PharmD

Blood (2019) 134 (Supplement_1): 905.

https://doi.org/10.1182/blood-2019-129690

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

Systemic Vascular Basement Membrane Markers Linked to Synovial Vascular Remodeling Are Bio-Markers of Hemarthrosis in Patients with Hemophilia

Srila Gopal, MD, Esther J Cooke, Jenny Y Zhou, Ilana Levin, Pamela Emery, Tina Manon- Jensen, Morten Karsdal, Annette von Drygalski, MD

Blood (2018) 132 (Supplement 1): 380.

https://doi.org/10.1182/blood-2018-99-112725

Published: 2018

Journal Articles

Chronic Kidney Disease in the U.S. Hemophilia Population: A Cohort Study

Blood (2017) 130 (Supplement 1): 2359.

https://doi.org/10.1182/blood.V130.Suppl_1.2359.2359

Published: 2017

Journal Articles

The CXCL12/CXCR4 and VEGF165/Nrp1 Pathways Have Differential Effects on Pathological Neo-Angiogenesis and Vascular Remodeling in Hemophilic Arthropathy

Tine L. Wyseure, PhD, Esther J. Cooke, PhD, Annette von Drygalski, MD, Laurent O. Mosnier, PhD

Blood (2017) 130 (Supplement 1): 180.

https://doi.org/10.1182/blood.V130.Suppl_1.180.180

Published: 2017

Journal Articles

Pocket Handheld Ultrasound As a Novel Point-of-Care Imaging Modality to Diagnose Bleeding in Hemophilic Joints

Jenny Y Zhou, MD, Katherine C. Rappazzo, Lena Volland, Bruno Steiner, Rebecca Kruse-Jarres, MD MPH, Doris V. Quon, MD PhD, Eric Y. Chang, MD, Annette von Drygalski, MD PharmD

Blood (2016) 128 (22): 2345.

https://doi.org/10.1182/blood.V128.22.2345.2345

Published: 2016

Journal Articles

Defective TAFI Activation in Hemophilia Worsens Joint Bleeding

Tine L Wyseure, PhD, Esther J Cooke, PhD, Paul J Declerck, PhD, Joost CM Meijers, PhD, Annette von Drygalski, MD PharmD, Laurent O Mosnier, PhD

Blood (2016) 128 (22): 3752.

https://doi.org/10.1182/blood.V128.22.3752.3752

Published: 2016

Journal Articles

Effects of Joint Blood Volume and Hemostasis Correction on Vascular Changes in the Joint after Induced Hemarthrosis in Hypocoagulable Mice

Esther J Cooke, PhD, Tine L Wyseure, PhD, Jenny Y Zhou, MD, Laurent O Mosnier, PhD, Annette von Drygalski, MD PharmD

Blood (2016) 128 (22): 2578.

https://doi.org/10.1182/blood.V128.22.2578.2578

Published: 2016

Journal Articles

"Hemophilia Vertical": Effects of Therapeutic Rock Climbing on Joint Health in Hemophilia Patients with Severe Arthropathies

Courtney Schroeder, Felix Kallenbach, Lena Volland, Richard FW Barnes, Colleen M Moran, Christine Geyer, Regina von Velsen, Annette von Drygalski, MD PharmD, Michaela Andrea Stemberger

Blood (2016) 128 (22): 4754.

https://doi.org/10.1182/blood.V128.22.4754.4754

Published: 2016

Journal Articles

Defective TAFI Activation in Hemophilia Exacerbates Vascular Remodeling in Hemophilic Arthropathy

Tine L Wyseure, PhD, Esther J Cooke, PhD, Paul J Declerck, PhD, Joost CM Meijers, PhD, Annette von Drygalski, MD PharmD, Laurent O Mosnier, PhD

Blood (2016) 128 (22): 82.

https://doi.org/10.1182/blood.V128.22.82.82

Published: 2016

Journal Articles

Vascular Remodeling Underlies Re-Bleeding in Hemophilic Arthropathy

Annette Von Drygalski, MD PharmD, Vikas Bhat, PhD, Merissa Olmer, MS, Shweta Joshi, PhD, Donald Durden, MD, Thomas J Cramer, PhD, Richard FW Barnes, PhD MPH, Scott T Ball, MD, Tudor H Hughes, MD, Mauricio Silva, MD, James V Luck, MD, Randy Moore, DC,RDMS, Laurent O. Mosnier, PhD

Blood (2015) 126 (23): 1038.

https://doi.org/10.1182/blood.V126.23.1038.1038

Published: 2015

NARROW

First Data from the Phase 3 HOPE-B Gene Therapy Trial: Efficacy and Safety of Etranacogene Dezaparvovec (AAV5-Padua hFIX variant; AMT-061) in Adults with Severe or Moderate-Severe Hemophilia B Treated Irrespective of Pre-Existing Anti-Capsid Neutralizing Antibodies

Lymphatic Remodeling Associated with Hemophilic Joint Bleeding in FVIII-Deficient Mice

Validity of Quantitative Measurements By the Joint Tissue Examination and Damage Exam (JADE) with Musculoskeletal Ultrasound for the Longitudinal Assessment of Hemophilic Arthropathy

Incidence of Venous Thromboembolic Events in Hospitalized COVID-19 Patients: A Systematic Review

Traumatic Bleeding in Mice Is Amplified By Iron Deficiency Anemia and Can be Mitigated By Tranexamic Acid Prophylaxis

Risk of Hypertension in Hemophilia Patients with a History of Inhibitors

Mechanisms of Iron Clearance from the Joint in FVIII-Deficient Mice after Induced Hemarthrosis

Prevention of Bleeding-Induced Vascular Abnormalities in the Hemophilic Mouse Joint By Increasing TAFI Levels or Inhibiting uPA Activity

Super factor Va, an Engineered Factor V Molecule, Effectively Reduces Bleeding and Acute Coagulopathy in Mouse Models of Trauma and Shock

Quantitative Measurements of Hemophilic Joint Tissues By Point-of-Care Musculoskeletal Ultrasound Are Associated with Clinical and Functional Joint Outcome Parameters

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

Systemic Vascular Basement Membrane Markers Linked to Synovial Vascular Remodeling Are Bio-Markers of Hemarthrosis in Patients with Hemophilia

Chronic Kidney Disease in the U.S. Hemophilia Population: A Cohort Study

The CXCL12/CXCR4 and VEGF165/Nrp1 Pathways Have Differential Effects on Pathological Neo-Angiogenesis and Vascular Remodeling in Hemophilic Arthropathy

Pocket Handheld Ultrasound As a Novel Point-of-Care Imaging Modality to Diagnose Bleeding in Hemophilic Joints

Defective TAFI Activation in Hemophilia Worsens Joint Bleeding

Effects of Joint Blood Volume and Hemostasis Correction on Vascular Changes in the Joint after Induced Hemarthrosis in Hypocoagulable Mice

"Hemophilia Vertical": Effects of Therapeutic Rock Climbing on Joint Health in Hemophilia Patients with Severe Arthropathies

Defective TAFI Activation in Hemophilia Exacerbates Vascular Remodeling in Hemophilic Arthropathy

Vascular Remodeling Underlies Re-Bleeding in Hemophilic Arthropathy

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American Society of Hematology

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American Society of Hematology

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