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Journal Articles

Activity of transgene-produced B-domain–deleted factor VIII in human plasma following AAV5 gene therapy

Steffen Rosen, Stefan Tiefenbacher, Mary Robinson, Mei Huang, Jaydeep Srimani, Donnie Mackenzie, Terri Christianson, K. John Pasi, Savita Rangarajan, Emily Symington, Adam Giermasz, Glenn F. Pierce, Benjamin Kim, Stephen J. Zoog, Christian Vettermann

Blood (2020) 136 (22): 2524–2534.

https://doi.org/10.1182/blood.2020005683

Published: 2020

Includes: Supplemental data

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

Examination and Validation of a Patient-Centric Joint Metric: "Problem Joint"; Empirical Evidence from the CHESS US Dataset

Tom Burke, Idaira Rodriguez Santana, PhD, Pratima Chowdary, MD MBBS, MRCP, FRCPATH, Randall Curtis, MBA, Kate Khair, PhD, Michael Laffan, DM, FRCP, FRCPath, Paul McLaughlin, MSc, MMACP, Declan Noone, MSc, Brian O'Mahony, John Pasi, MB, ChB, PhD FRCP, FRCPath, FRCPCH, Mark W Skinner, JD, Jamie O'Hara, MSc

Blood (2020) 136 (Supplement 1): 25–26.

https://doi.org/10.1182/blood-2020-140950

Published: 2020

Journal Articles

Long-Term Durability, Safety and Efficacy of Fitusiran Prophylaxis in People with Hemophilia a or B, with or without Inhibitors - Results from the Phase II Study

Steven W. Pipe, MD, John Pasi, MB, ChB, PhD FRCP, FRCPath, FRCPCH, Toshko Lissitchkov, Margaret V. Ragni, MDMPH, Claude Négrier, Qifeng Yu, Stacey Poloskey, Baisong Mei, Shauna R. Andersson

Blood (2020) 136 (Supplement 1): 3–4.

https://doi.org/10.1182/blood-2020-136501

Published: 2020

Journal Articles

First-in-Human Phase 1/2 Clinical Trial of SIG-001, an Innovative Shielded Cell Therapy Platform, for Hemophilia Α

Amy D Shapiro, MD, Barbara A. Konkle, MD, Stacy E. Croteau, MD, Wolfgang A. Miesbach, MD, Charles Richard Morris Hay, MDFRCP,FRCPath, Rashid Kazmi, MBBS, Marina Mihova, Savita Rangarajan, MD MBBS, John Pasi, MB, ChB, PhD FRCP, FRCPath, FRCPCH

Blood (2020) 136 (Supplement 1): 8.

https://doi.org/10.1182/blood-2020-141381

Published: 2020

Journal Articles

Longitudinal Assessment of Thrombin Generation in Patients with Hemophilia Receiving Fitusiran Prophylaxis: Phase II Study Results

Claude Négrier, Margaret V. Ragni, MDMPH, John Pasi, MB, ChB, PhD FRCP, FRCPath, FRCPCH, Steven W. Pipe, MD, Gili Kenet, Savita Rangarajan, MBBS,MDFRCP,FRCPath, Salim Kichou, Baisong Mei, Shauna R. Andersson

Blood (2020) 136 (Supplement 1): 36–37.

https://doi.org/10.1182/blood-2020-136536

Published: 2020

Journal Articles

Novel manifestations of immune dysregulation and granule defects in gray platelet syndrome

Matthew C. Sims, Louisa Mayer, Janine H. Collins, Tadbir K. Bariana, Karyn Megy, Cecile Lavenu-Bombled, Denis Seyres, Laxmikanth Kollipara, Frances S. Burden, Daniel Greene, Dave Lee, Antonio Rodriguez-Romera, Marie-Christine Alessi, William J. Astle, Wadie F. Bahou, Loredana Bury, Elizabeth Chalmers, Rachael Da Silva, Erica De Candia, Sri V. V. Deevi, Samantha Farrow, Keith Gomez, Luigi Grassi, Andreas Greinacher, Paolo Gresele, Dan Hart, Marie-Françoise Hurtaud, Anne M. Kelly, Ron Kerr, Sandra Le Quellec, Thierry Leblanc, Eva B. Leinøe, Rutendo Mapeta, Harriet McKinney, Alan D. Michelson, Sara Morais, Diane Nugent, Sofia Papadia, Soo J. Park, John Pasi, Gian Marco Podda, Man-Chiu Poon, Rachel Reed, Mallika Sekhar, Hanna Shalev, Suthesh Sivapalaratnam, Orna Steinberg-Shemer, Jonathan C. Stephens, Robert C. Tait, Ernest Turro, John K. M. Wu, Barbara Zieger, NIHR BioResource, Taco W. Kuijpers, Anthony D. Whetton, Albert Sickmann, Kathleen Freson, Kate Downes, Wendy N. Erber, Mattia Frontini, Paquita Nurden, Willem H. Ouwehand, Remi Favier, Jose A. Guerrero

Blood (2020) 136 (17): 1956–1967.

https://doi.org/10.1182/blood.2019004776

Published: 2020

Includes: Supplemental data

Journal Articles

The Role of Antithrombin Lowering in Patients with Hemophilia: Hemostatic Control Pre- and Post-Fitusiran Dosing Interruption

Margaret V. Ragni, MD MPH, Pencho Georgiev, MD, Michael Desmond Creagh, Toshko Jelev Lissitchkov, MD, Steve K. Austin, BMedSci, MBBS, Charles Richard Morris Hay, MDFRCP, FRCPath, Inga Hegemann, MD, Syed Rashid Saeed Kazmi, MBBS, Pratima Chowdary, MD, Savita Rangarajan, MBBS, MD, Meredith C. Foster, ScD, Qifeng Yu, PhD, Craig Benson, MD MS, Kate Madigan, MD, Huy Van Nguyen, PharmD, Shariq Ali, PhD, Ketaki Kadam, MS, Pallavi Jain, PharmD, K. John Pasi, MB, ChB, PhD FRCP, FRCPath, FRCPCH

Blood (2018) 132 (Supplement 1): 2477.

https://doi.org/10.1182/blood-2018-99-112998

Published: 2018

Journal Articles

Gene Therapy in Hemophilia: An Assessment of Hematologists' Knowledge Gaps and Attitudes

Simi Hurst, PhD, Charlotte Warren, K. John Pasi, MB, ChB, PhD FRCP, FRCPath, FRCPCH

Blood (2018) 132 (Supplement 1): 3485.

https://doi.org/10.1182/blood-2018-99-119116

Published: 2018

Journal Articles

B-YOND Final Results Confirm Established Safety, Sustained Efficacy, and Extended Dosing Interval for Up to 4 Years of Treatment With rFIXFc in Previously Treated Subjects With Severe Hemophilia B

Margaret Ragni, Roshni Kulkarni, K. John Pasi, MB, ChB, PhD FRCP, FRCPath, FRCPCH, Kathelijn Fischer, Johnny Mahlangu, Amy Shapiro, Beatrice Nolan, Johannes Oldenburg, Tadashi Matsushita, Annemieke Willemze, Huixing Yuan, Dan Rudin

Blood (2018) 132 (Supplement 1): 1214.

https://doi.org/10.1182/blood-2018-99-110100

Published: 2018

Journal Articles

ASPIRE Final Results Confirm Established Safety and Sustained Efficacy for Up to 4 Years of Treatment With rFVIIIFc in Previously Treated Subjects With Severe Hemophilia A

Beatrice Nolan, Johnny Mahlangu, Guy Young, Barbara A Konkle, K. John Pasi, MB, ChB, PhD FRCP, FRCPath, FRCPCH, Johannes Oldenburg, MD PhD, Keiji Nogami, MDPhD, Nikola Tripkovic, Huixing Yuan, Dan Rudin

Blood (2018) 132 (Supplement 1): 1192.

https://doi.org/10.1182/blood-2018-99-118586

Published: 2018

Journal Articles

Achievement of Normal Circulating Factor VIII Activity Following Bmn 270 AAV5-FVIII Gene Transfer: Interim, Long-Term Efficacy and Safety Results from a Phase 1/2 Study in Patients with Severe Hemophilia a

K. John Pasi, Savita Rangarajan, Benjamin Kim, Will Lester, David Perry, Bella Madan, Fatemeh Tavakkoli, Ke Yang, Glenn F. Pierce, Wing Yen Wong

Blood (2017) 130 (Supplement 1): 603.

https://doi.org/10.1182/blood.V130.Suppl_1.603.603

Published: 2017

Journal Articles

Clinical Outcomes of 14-Day Prophylaxis with rFIXFc: Longitudinal Analysis of the B-LONG and B-YOND Study

Amy D. Shapiro, MD, Margareth C Ozelo, MD PhD, K John Pasi, Jingyuan Feng, Bent Winding, Chris Barnowski, Roshni Kulkarni

Blood (2017) 130 (Supplement 1): 3667.

https://doi.org/10.1182/blood.V130.Suppl_1.3667.3667

Published: 2017

Journal Articles

Perioperative Management in Patients with Hemophilia Receiving Fitusiran, an Investigational RNAi Therapeutic Targeting Antithrombin for the Treatment of Hemophilia

Claude Negrier, MD, Margaret V. Ragni, MD MPH, Pencho Georgiev, MD, Toshko Jelev Lissitchkov, MD PhD, Huy Van Nguyen, PharmD, Kate Madigan, K John Pasi

Blood (2017) 130 (Supplement 1): 2366.

https://doi.org/10.1182/blood.V130.Suppl_1.2366.2366

Published: 2017

Journal Articles

Fitusiran, an Investigational RNAi Therapeutic Targeting Antithrombin for the Treatment of Hemophilia: Updated Results from a Phase 1 and Phase 1/2 Extension Study in Patients with Inhibitors

K John Pasi, Pencho Georgiev, Tim Mant, Toshko Lissitchkov, Michael Desmond Creagh, David Bevan, Steve Austin, Charles R Hay, Inga Hegemann, Rashid Kazmi, Pratima Chowdary, Margaret V. Ragni, MD, Chang-Heok Soh, Akin Akinc, Angela M. Partisano, Benny Sorenson, Savita Rangarajan

Blood (2016) 128 (22): 1397.

https://doi.org/10.1182/blood.V128.22.1397.1397

Published: 2016

Journal Articles

Longitudinal Analysis of Long-Term Safety and Efficacy of Recombinant Factor VIII Fc Fusion Protein (rFVIIFc) in Adults/Adolescents with Severe Hemophilia a

Barbara Konkle, MD, K John Pasi, David J Perry, Johnny Mahlangu, Savita Rangarajan, Simon A Brown, Hideji Hanabusa, Ingrid Pabinger, Lynda M Cristiano, Elisa Tsao, Bent Winding, Desilu Glazebrook, Stefan Lethagen, Shannon Jackson

Blood (2016) 128 (22): 1413.

https://doi.org/10.1182/blood.V128.22.1413.1413

Published: 2016

Journal Articles

Longitudinal Analysis of Long-Term Safety and Efficacy of Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) in Previously Treated Children with Severe Hemophilia a

Guy Young, Raina Liesner, K John Pasi, Beatrice Nolan, Stefan Lethagen, Lynda M Cristiano, Elisa Tsao, Bent Winding, Johnny Mahlangu

Blood (2016) 128 (22): 1414.

https://doi.org/10.1182/blood.V128.22.1414.1414

Published: 2016

Journal Articles

Clinical Outcomes in Adults/Adolescents with Hemophilia B Treated Long Term with Recombinant Factor IX Fc Fusion Protein (rFIXFc) Prophylaxis: Interim Results of the B-Yond Extension Study

Tadashi Matsushita, Johnny Mahlangu, Amy D Shapiro, K John Pasi, Margaret V. Ragni, MD, Margareth C Ozelo, Johannes Oldenburg, Prof, Ross I Baker, Huixing Yuan, Alejandra Ramirez-Santiago, Francesca Ferrante, Stefan Lethagen

Blood (2016) 128 (22): 1416.

https://doi.org/10.1182/blood.V128.22.1416.1416

Published: 2016

Journal Articles

Long-Term Efficacy and Quality of Life with Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) Prophylaxis in Pediatric, Adolescent, and Adult Subjects with Target Joints and Severe Hemophilia a

Michael Wang, MD, K John Pasi, Ingrid Pabinger, Prof, Bryce A. Kerlin, Roshni Kulkarni, MD, Beatrice Nolan, Raina Liesner, Simon A Brown, Hideji Hanabusa, Elisa Tsao, Bent Winding, Stefan Lethagen, Nisha Jain

Blood (2016) 128 (22): 3791.

https://doi.org/10.1182/blood.V128.22.3791.3791

Published: 2016

Journal Articles

Fitusiran, an Investigational RNAi Therapeutic Targeting Antithrombin for the Treatment of Hemophilia: Updated Results from a Phase 1 and Phase 1/2 Extension Study in Patients without Inhibitors

Margaret V. Ragni, MD, Pencho Georgiev, Tim Mant, Michael Desmond Creagh, Toshko Lissitchkov, David Bevan, Steve Austin, Charles R Hay, Inga Hegemann, Rashid Kazmi, Pratima Chowdary, Savita Rangarajan, Chang-Heok Soh, Akin Akinc, Angela M. Partisano, Benny Sorenson, K John Pasi

Blood (2016) 128 (22): 2572.

https://doi.org/10.1182/blood.V128.22.2572.2572

Published: 2016

NARROW

Activity of transgene-produced B-domain–deleted factor VIII in human plasma following AAV5 gene therapy

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

Examination and Validation of a Patient-Centric Joint Metric: "Problem Joint"; Empirical Evidence from the CHESS US Dataset

Long-Term Durability, Safety and Efficacy of Fitusiran Prophylaxis in People with Hemophilia a or B, with or without Inhibitors - Results from the Phase II Study

First-in-Human Phase 1/2 Clinical Trial of SIG-001, an Innovative Shielded Cell Therapy Platform, for Hemophilia Α

Longitudinal Assessment of Thrombin Generation in Patients with Hemophilia Receiving Fitusiran Prophylaxis: Phase II Study Results

Novel manifestations of immune dysregulation and granule defects in gray platelet syndrome

The Role of Antithrombin Lowering in Patients with Hemophilia: Hemostatic Control Pre- and Post-Fitusiran Dosing Interruption

Gene Therapy in Hemophilia: An Assessment of Hematologists' Knowledge Gaps and Attitudes

B-YOND Final Results Confirm Established Safety, Sustained Efficacy, and Extended Dosing Interval for Up to 4 Years of Treatment With rFIXFc in Previously Treated Subjects With Severe Hemophilia B

ASPIRE Final Results Confirm Established Safety and Sustained Efficacy for Up to 4 Years of Treatment With rFVIIIFc in Previously Treated Subjects With Severe Hemophilia A

Achievement of Normal Circulating Factor VIII Activity Following Bmn 270 AAV5-FVIII Gene Transfer: Interim, Long-Term Efficacy and Safety Results from a Phase 1/2 Study in Patients with Severe Hemophilia a

Clinical Outcomes of 14-Day Prophylaxis with rFIXFc: Longitudinal Analysis of the B-LONG and B-YOND Study

Perioperative Management in Patients with Hemophilia Receiving Fitusiran, an Investigational RNAi Therapeutic Targeting Antithrombin for the Treatment of Hemophilia

Fitusiran, an Investigational RNAi Therapeutic Targeting Antithrombin for the Treatment of Hemophilia: Updated Results from a Phase 1 and Phase 1/2 Extension Study in Patients with Inhibitors

Longitudinal Analysis of Long-Term Safety and Efficacy of Recombinant Factor VIII Fc Fusion Protein (rFVIIFc) in Adults/Adolescents with Severe Hemophilia a

Longitudinal Analysis of Long-Term Safety and Efficacy of Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) in Previously Treated Children with Severe Hemophilia a

Clinical Outcomes in Adults/Adolescents with Hemophilia B Treated Long Term with Recombinant Factor IX Fc Fusion Protein (rFIXFc) Prophylaxis: Interim Results of the B-Yond Extension Study

Long-Term Efficacy and Quality of Life with Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) Prophylaxis in Pediatric, Adolescent, and Adult Subjects with Target Joints and Severe Hemophilia a

Fitusiran, an Investigational RNAi Therapeutic Targeting Antithrombin for the Treatment of Hemophilia: Updated Results from a Phase 1 and Phase 1/2 Extension Study in Patients without Inhibitors

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American Society of Hematology

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American Society of Hematology

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