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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

Hemophilia Natural History Study (ATHN 7): Baseline Characteristics, Adverse Events, and Self-Reported Health Status of Individuals with Hemophilia a and B

Tyler W. Buckner, MDMSc, Shannon L Carpenter, MD, Stacy E. Croteau, MD, Adam Cuker, MDMS, Nabil Daoud, MA, Christine L Kempton, MDMSc, Lynn M Malec, MD, Thomas W. McLean, MD, Leslie J. Raffini, MD, Janice M Staber, MD, Michael Wang, MD, Crystal Watson, BS, Ayesha Zia, MD, Michael Recht, MD PhD

Blood (2020) 136 (Supplement 1): 2–3.

https://doi.org/10.1182/blood-2020-137696

Published: 2020

Journal Articles

Results of Genetic Analysis of 11,341 Participants Enrolled in the My Life, Our Future (MLOF) Hemophilia Genotyping Initiative

Jill M Johnsen, MD, Shelley N Fletcher, Angela Dove, Haley McCracken, Beth K Martin, Martin Kircher, Neil C Josephson, MD, Jay Shendure, Sarah Ruuska, MPH, Leonard A. Valentino, MD, Glenn Pierce, MD PhD, Crystal Watson, BS, Dunlei Cheng, PhD, Michael Recht, MD PhD, Barbara A. Konkle, MD

Blood (2020) 136 (Supplement 1): 19.

https://doi.org/10.1182/blood-2020-140649

Published: 2020

Journal Articles

Dosing, Patient Satisfaction and Other Patient-Reported Outcomes after Switching to Rurioctocog Alfa Pegol in Athn 2: A Longitudinal, Observational Study of Previously Treated Hemophilia Patients Switching Coagulation Replacement Factor Products

Janna M. Journeycake, MD, Michael Recht, MD PhD, Christine Guelcher, Hemostasis RN-BC, MS, PPCNP-BC, Ellis J. Neufeld, Margaret V. Ragni, MDMPH, Robert F. Sidonio, Jr., MDMSc, Cliff Takemoto, Michael D Tarantino, MD, Dunlei Cheng, PhD, Susan U. Lattimore, Jorge Caicedo, Michael Denne, PharmD

Blood (2020) 136 (Supplement 1): 17–18.

https://doi.org/10.1182/blood-2020-138898

Published: 2020

Journal Articles

In Vitro Evaluation of Thrombin Generation of Eptacog Beta (Factor VIIa, Recombinant) and Emicizumab in Congenital Hemophilia Α Plasma with and without Inhibitors

Steven W. Pipe, MD, Michael Recht, MD PhD, Michael U. Callaghan, MD, Robert F. Sidonio, Jr., MDMSc, Jerry Grandoni, PhD, Veronique Duretz, Daniel Bonzo, PhD, Jean Luc Plantier, PhD, Sean Evans, PhD RPh, Ian S Mitchell, PhD, Cedric Hermans, MDMRCP,PhD

Blood (2020) 136 (Supplement 1): 41–42.

https://doi.org/10.1182/blood-2020-136430

Published: 2020

Journal Articles

Etranacogene Dezaparvovec (AAV5-Padua hFIX variant), an Enhanced Vector for Gene Transfer in Adults with Severe or Moderate-Severe Hemophilia B: Two Year Data from a Phase 2b Trial

Annette von Drygalski, Adam Giermasz, MD PhD, Giancarlo Castaman, MD, Nigel S. Key, MD, Susan U. Lattimore, Frank W.G. Leebeek, MD PhD, Wolfgang A. Miesbach, MD, Michael Recht, MD PhD, Esteban Gomez, MD, Robert Gut, Steven W. Pipe, MD

Blood (2020) 136 (Supplement 1): 13.

https://doi.org/10.1182/blood-2020-139295

Published: 2020

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

Early Prophylaxis Provides Continued Joint Protection in Severe Hemophilia A: Results of the Joint Outcome Continuation Study

Beth Boulden Warren, MD MS, Dianne Thornhill, PhD, Jill Stein, MD, Michael Fadell, MD, Sharon Funk, DPT, Heidi D. Lane, DPT, Carolyn M. Bennett, MD MSc, Amy Shapiro, Michael Recht, MD PhD, Marilyn J Manco-Johnson, MD

Blood (2018) 132 (Supplement 1): 382.

https://doi.org/10.1182/blood-2018-99-117413

Published: 2018

Journal Articles

Data Coming out of the Human Inhibitor PUP Study (HIPS) Reveal 4 Subgroups of Patients with Distinct Antibody Signatures

Blood (2018) 132 (Supplement 1): 3774.

https://doi.org/10.1182/blood-2018-99-115979

Published: 2018

Journal Articles

Adeno-Associated Mediated Gene Transfer for Hemophilia B:8 Year Follow up and Impact of Removing "Empty Viral Particles" on Safety and Efficacy of Gene Transfer

Amit C Nathwani, MD PhD, Ulrike Reiss, MD, Edward Tuddenham, MD, Pratima Chowdary, MD, Jenny McIntosh, PhD, Anne Riddell, Jun Pie, Johnny N Mahlangu, MD, Michael Recht, MD PhD, Yu-Min Shen, MD, Kathleen G. Halka, MD, Michael M Meagher, PhD, Arthur W. Nienhuis, MD, Andrew M. Davidoff, MD, Sarah Mangles, Chris L Morton, Zhou Junfang, Vlad Calin Radulescu, MD

Blood (2018) 132 (Supplement 1): 491.

https://doi.org/10.1182/blood-2018-99-118334

Published: 2018

Journal Articles

Longitudinal Antibody Signatures Following FVIII Replacement Therapy in Previously Untreated Patients with Severe Hemophilia Α- New Insights from the Hemophilia Inhibitor PUP Study (HIPS)

Bagirath Gangadharan, PhD, Birgit Reipert, Fritz Scheiflinger, PhD, Joel Bowen, Elizabeth Donnachie, PhD, Karin Fijn van Draat, Ralph A. Gruppo, MD, Jenny Klintman, MD PhD, Christoph Male, MD, Catherine E. McGuinn, MD, Shannon L. Meeks, MD, Michael Recht, MD PhD, Margaret V. Ragni, MD MPH, Hassan M. Yaish, MD, Amy D. Shapiro, MD, Donald L Yee, MD, Vlad Calin Radulescu, MD, Elena Santagostino, Deborah Brown, MD

Blood (2017) 130 (Supplement 1): 88.

https://doi.org/10.1182/blood.V130.Suppl_1.88.88

Published: 2017

Journal Articles

Appearance of High-Affinity Antibodies Precedes Clinical Diagnosis of FVIII Inhibitors - Preliminary Analysis from the Hemophilia Inhibitor PUP Study (HIPS)

Birgit M Reipert, PhD, Bagirath Gangadharan, PhD, Christoph J Hofbauer, PhD, Fritz Scheiflinger, PhD, Joel Bowen, Elizabeth Donnachie, PhD, Karen Fijvandraat, MD, Ralph A. Gruppo, MD, Jenny Klintman, MD PhD, Christoph Male, MD, Catherine E. McGuinn, MD, Shannon L. Meeks, MD, Michael Recht, MD PhD, M V Ragni, MD, Hassan M Yaish, Elena Santagostino, MD PhD, Deborah L Brown, MD

Blood (2016) 128 (22): 328.

https://doi.org/10.1182/blood.V128.22.328.328

Published: 2016

Journal Articles

Linear and Logistic Regression Models of Patient-Reported Outcomes and Patient Characteristics in US Adults with Hemophilia from the Pain, Functional Impairment, and Quality of Life (P-FiQ) Study

Katharine Batt, MD MSc, Michael Recht, MD PhD, Michael Wang, MD, Doris V. Quon, MD PhD, Lisa N Boggio, MD MS, Craig M. Kessler, MD, Tyler W. Buckner, MD MSc, Anne T. Neff, MD, Neeraj N Iyer, PhD, David L Cooper, MD MBA, Christine L Kempton, MD MSc

Blood (2016) 128 (22): 252.

https://doi.org/10.1182/blood.V128.22.252.252

Published: 2016

Journal Articles

Identification of Qualitative Platelet Disorders in Adolescent Women with Heavy Menstrual Bleeding

Kristina M. Haley, DO, Susan Lattimore, RN, Cara McDavitt, Ayesha Khader, Colin Boehnlein, Sandra Baker-Groberg, PhD, Anh Ngo, Owen J.T. McCarty, PhD, Michael Recht, MD PhD

Blood (2016) 128 (22): 4922.

https://doi.org/10.1182/blood.V128.22.4922.4922

Published: 2016

Journal Articles

Safety and Efficacy of Turoctocog Alfa in Prevention and on-Demand Treatment of Bleeding Episodes in Patients with Hemophilia A

Dragana Janic, MD PhD, Irina Matytsina, MD, Mudi Misgav, Johannes Oldenburg, Prof, Margareth C Ozelo, Michael Recht, MD PhD, Lars Korsholm, Aleksandar Savic, Elena Santagostino, MD PhD

Blood (2016) 128 (22): 3790.

https://doi.org/10.1182/blood.V128.22.3790.3790

Published: 2016

Journal Articles

Bleeding Events and Safety Outcomes in Patients with Hemophilia a with Inhibitors: A Prospective, Multicenter, Non-Interventional Study

Johnny Mahlangu, Johannes Oldenburg, Prof, Michael U Callaghan, MD, Midori Shima, MD PhD, Elena Santagostino, MD PhD, Michaela Lehle, PhD, Marianne Uguen, MSc, Ceri Hirst, PhD, Maggie Moore, RN, Michael Recht, MD PhD, Claudia Garcia, MD, Renchi Yang, MD PhD, Rebecca Kruse-Jarres, MD MPH

Blood (2016) 128 (22): 3800.

https://doi.org/10.1182/blood.V128.22.3800.3800

Published: 2016

Journal Articles

Genes Influencing the Development and Severity of Chronic ITP Identified through Whole Exome Sequencing

Jenny M. Despotovic, DO, MS, Linda M. Polfus, PhD, Jonathan M. Flanagan, PhD, Carolyn M. Bennett, Michele P Lambert, MD, Cindy Neunert, MD, Manjusha Kumar, MD, Robert J. Klaassen, MD FRCP, Courtney Thornburg, MD MS, Michael Jeng, MD, Michael Recht, MD PhD, Susan E. Kirk, PA-C, Alexis A. Thompson, MD, Diane J. Nugent, MD, Ellis J. Neufeld, MD PhD, James B. Bussel, MD, Eric Boerwinkle, PhD, Rachael F. Grace, MD

Blood (2015) 126 (23): 73.

https://doi.org/10.1182/blood.V126.23.73.73

Published: 2015

Journal Articles

The Clinical Associations and Outcomes of Patients Admitted with Acquired Hemophilia; A Review of the 2012 National Inpatient Sample

Joseph Shatzel, MD, Sharl Azar, MD, Nathanael David Gay, MD, Jai Luo, MD, Thomas G. Deloughery, MD, Michael Recht, MD PhD, Jason A. Taylor, MD PhD

Blood (2015) 126 (23): 1091.

https://doi.org/10.1182/blood.V126.23.1091.1091

Published: 2015

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

Non-Factor VIII Coagulation Proteins Fail to Predict Joint Outcome in Severe Hemophilia a

Beth Boulden Warren, MD MS, Linda Jacobsen, BS, Elizabeth Villalobos-Menuey, BS, Thomas C. Abshire, MD, Amy D Shapiro, MD, Michael Recht, MD PhD, Deborah Brown, MD, Cindy A. Leissinger, MD, Christine L. Kempton, MD MSc, Heidi Lane, PT, DPT, PCS, Diane J. Nugent, MD, Marilyn J. Manco-Johnson, MD

Blood (2015) 126 (23): 2289.

https://doi.org/10.1182/blood.V126.23.2289.2289

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

Hemophilia Natural History Study (ATHN 7): Baseline Characteristics, Adverse Events, and Self-Reported Health Status of Individuals with Hemophilia a and B

Results of Genetic Analysis of 11,341 Participants Enrolled in the My Life, Our Future (MLOF) Hemophilia Genotyping Initiative

Dosing, Patient Satisfaction and Other Patient-Reported Outcomes after Switching to Rurioctocog Alfa Pegol in Athn 2: A Longitudinal, Observational Study of Previously Treated Hemophilia Patients Switching Coagulation Replacement Factor Products

In Vitro Evaluation of Thrombin Generation of Eptacog Beta (Factor VIIa, Recombinant) and Emicizumab in Congenital Hemophilia Α Plasma with and without Inhibitors

Etranacogene Dezaparvovec (AAV5-Padua hFIX variant), an Enhanced Vector for Gene Transfer in Adults with Severe or Moderate-Severe Hemophilia B: Two Year Data from a Phase 2b Trial

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

Early Prophylaxis Provides Continued Joint Protection in Severe Hemophilia A: Results of the Joint Outcome Continuation Study

Data Coming out of the Human Inhibitor PUP Study (HIPS) Reveal 4 Subgroups of Patients with Distinct Antibody Signatures

Adeno-Associated Mediated Gene Transfer for Hemophilia B:8 Year Follow up and Impact of Removing "Empty Viral Particles" on Safety and Efficacy of Gene Transfer

Longitudinal Antibody Signatures Following FVIII Replacement Therapy in Previously Untreated Patients with Severe Hemophilia Α- New Insights from the Hemophilia Inhibitor PUP Study (HIPS)

Appearance of High-Affinity Antibodies Precedes Clinical Diagnosis of FVIII Inhibitors - Preliminary Analysis from the Hemophilia Inhibitor PUP Study (HIPS)

Linear and Logistic Regression Models of Patient-Reported Outcomes and Patient Characteristics in US Adults with Hemophilia from the Pain, Functional Impairment, and Quality of Life (P-FiQ) Study

Identification of Qualitative Platelet Disorders in Adolescent Women with Heavy Menstrual Bleeding

Safety and Efficacy of Turoctocog Alfa in Prevention and on-Demand Treatment of Bleeding Episodes in Patients with Hemophilia A

Bleeding Events and Safety Outcomes in Patients with Hemophilia a with Inhibitors: A Prospective, Multicenter, Non-Interventional Study

Genes Influencing the Development and Severity of Chronic ITP Identified through Whole Exome Sequencing

The Clinical Associations and Outcomes of Patients Admitted with Acquired Hemophilia; A Review of the 2012 National Inpatient Sample

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

Non-Factor VIII Coagulation Proteins Fail to Predict Joint Outcome in Severe Hemophilia a

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American Society of Hematology

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American Society of Hematology

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