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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

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

Single 225μg/Kg Dose Treatment with Eptacog Beta (Factor VIIa, Recombinant) Results in Rapid Hemostasis in Joint Bleeds for Persons with Hemophilia Α or Β with Inhibitors: A PERSEPT1 Subset Analysis

Janna M. Journeycake, MD, Cedric Hermans, MDMRCP,PhD, Jonathan M. Ducore, MD MPH, Miguel A. Escobar, MD, Guy Young, MD, Michael Wang, MD, Doris Quon, MD PhD, W Allan Alexander, MD, Ian S Mitchell, PhD, Ahmad Al-Sabbagh, MD, Daniel Bonzo, PhD

Blood (2020) 136 (Supplement 1): 2–3.

https://doi.org/10.1182/blood-2020-136443

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

PERSEPT3: A Phase 3 Clinical Trial to Evaluate the Hemostatic Efficacy of Eptacog Beta (Recombinant Human FVIIa) in Perioperative Care in Subjects with Hemophilia Α or B with Inhibitors

Miguel A. Escobar, MD, Doris Quon, MD PhD, Cindy A. Leissinger, MD, James V Luck, Jr., MD, Ahmad Al-Sabbagh, MD, Daniel Bonzo, PhD, Ian S Mitchell, PhD, W Allan Alexander, MD, Cedric Hermans, MDMRCP,PhD

Blood (2020) 136 (Supplement 1): 34–36.

https://doi.org/10.1182/blood-2020-136448

Published: 2020

Journal Articles

A Phase IV, Multicenter, Open-Label Study of Emicizumab Prophylaxis in Persons with Hemophilia a with or without FVIII Inhibitors Undergoing Minor Surgical Procedures

Miguel A. Escobar, MD, Amy Dunn, Doris Quon, MD PhD, Ben Trzaskoma, Lucy Lee, Richard H Ko, Shannon L Carpenter, MD

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

https://doi.org/10.1182/blood-2020-134906

Published: 2020

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

BIVV001: The First Investigational Factor VIII Therapy to Break Through the VWF Ceiling in Hemophilia A, with Potential for Extended Protection for One Week or Longer

Barbara A Konkle, Amy Shapiro, Doris Quon, Janice Staber, Takashi Suzuki, MD PhD, Stacey Poloskey, Kara Rice, Suresh Katragadda, Dan Rudin, Joachim Fruebis

Blood (2018) 132 (Supplement 1): 636.

https://doi.org/10.1182/blood-2018-99-113575

Published: 2018

Journal Articles

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

Blood (2018) 132 (Supplement 1): 2479.

https://doi.org/10.1182/blood-2018-99-119637

Published: 2018

Journal Articles

Real-World Data on the Use of rFIXFc in Subjects With Hemophilia B for Up to 3.7 Years Demonstrates Improved Bleed Control and Adherence With Reduced Treatment Burden

Amy Shapiro, Ateefa Chaudhury, Nisha Jain, Elisa Tsao, Chris Barnowski, Jing Feng, Doris Quon

Blood (2018) 132 (Supplement 1): 2493.

https://doi.org/10.1182/blood-2018-99-116909

Published: 2018

Journal Articles

Clinical Outcomes of Weekly Prophylaxis with rFVIIIFc: Longitudinal Analysis of the A-LONG and ASPIRE Study Population

Amy D. Shapiro, MD, Alok Srivastava, MD, Margaret V. Ragni, MD MPH, Ingrid Pabinger, MD, Stefan Lethagen, Elisa Tsao, Desilu Glazebrook, Doris Quon

Blood (2017) 130 (Supplement 1): 2368.

https://doi.org/10.1182/blood.V130.Suppl_1.2368.2368

Published: 2017

Journal Articles

Impact of Pain and Functional Impairment in US Adult People with Hemophilia (PWH): Patient-Reported Outcomes and Musculoskeletal Evaluation in the Pain, Functional Impairment, and Quality of Life (P-FiQ) Study

Christine L Kempton, MD MSc, Michael Recht, MD PhD, Anne Neff, MD, Michael Wang, MD, Tyler W. Buckner, MD MSc, Amit Soni, MD, Doris Quon, MD PhD, Michelle Witkop, DNP, FNP-BC, Lisa Boggio, MD, David L Cooper, MD MBA

Blood (2015) 126 (23): 39.

https://doi.org/10.1182/blood.V126.23.39.39

Published: 2015

Journal Articles

A Cross-Sectional Analysis of Cardiovascular Disease in the Hemophilia Population

Blood (2015) 126 (23): 761.

https://doi.org/10.1182/blood.V126.23.761.761

Published: 2015

Journal Articles

Safety and prolonged activity of recombinant factor VIII Fc fusion protein in hemophilia A patients

Jerry S. Powell, Neil C. Josephson, Doris Quon, Margaret V. Ragni, Gregory Cheng, Ella Li, Haiyan Jiang, Lian Li, Jennifer A. Dumont, Jaya Goyal, Xin Zhang, Jurg Sommer, Justin McCue, Margaret Barbetti, Alvin Luk, Glenn F. Pierce

Blood (2012) 119 (13): 3031–3037.

https://doi.org/10.1182/blood-2011-09-382846

Published: 2012

Includes: Supplemental data

Journal Articles

Supplemental Alpha-2-Antiplasmin Prevents Plasmin-Induced Hemorrhage.

Daphne Stewart, MD, Victor J. Marder, MD, Mansze Kong, MPH, Doris Quon, MD, PhD, Scott A. Cook, PhD, Gary Jesmok, PhD, Peter Radtke, PhD, George A. Baumbach, PhD

Blood (2004) 104 (11): 1864.

https://doi.org/10.1182/blood.V104.11.1864.1864

Published: 2004

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

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

Single 225μg/Kg Dose Treatment with Eptacog Beta (Factor VIIa, Recombinant) Results in Rapid Hemostasis in Joint Bleeds for Persons with Hemophilia Α or Β with Inhibitors: A PERSEPT1 Subset Analysis

Risk of Hypertension in Hemophilia Patients with a History of Inhibitors

PERSEPT3: A Phase 3 Clinical Trial to Evaluate the Hemostatic Efficacy of Eptacog Beta (Recombinant Human FVIIa) in Perioperative Care in Subjects with Hemophilia Α or B with Inhibitors

A Phase IV, Multicenter, Open-Label Study of Emicizumab Prophylaxis in Persons with Hemophilia a with or without FVIII Inhibitors Undergoing Minor Surgical Procedures

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

BIVV001: The First Investigational Factor VIII Therapy to Break Through the VWF Ceiling in Hemophilia A, with Potential for Extended Protection for One Week or Longer

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

Real-World Data on the Use of rFIXFc in Subjects With Hemophilia B for Up to 3.7 Years Demonstrates Improved Bleed Control and Adherence With Reduced Treatment Burden

Clinical Outcomes of Weekly Prophylaxis with rFVIIIFc: Longitudinal Analysis of the A-LONG and ASPIRE Study Population

Impact of Pain and Functional Impairment in US Adult People with Hemophilia (PWH): Patient-Reported Outcomes and Musculoskeletal Evaluation in the Pain, Functional Impairment, and Quality of Life (P-FiQ) Study

A Cross-Sectional Analysis of Cardiovascular Disease in the Hemophilia Population

Safety and prolonged activity of recombinant factor VIII Fc fusion protein in hemophilia A patients

Supplemental Alpha-2-Antiplasmin Prevents Plasmin-Induced Hemorrhage.

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American Society of Hematology

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ASH Publications

American Society of Hematology

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