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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

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

Journal Articles

Long-Term Safety and Efficacy of rFVIIIFc in Adults and Adolescents with Severe Hemophilia A: A Longitudinal Analysis of A-LONG and ASPIRE

K John Pasi, David J Perry, Johnny Mahlangu, Barbara A Konkle, MD, Savita Rangarajan, Simon Brown, Hideji Hanabusa, Shannon Jackson, Ingrid Pabinger, Lynda M Cristiano, Yingwen Dong, Elisa Tsao, Glenn F Pierce, Geoffrey Allen

Blood (2015) 126 (23): 1087.

https://doi.org/10.1182/blood.V126.23.1087.1087

Published: 2015

Journal Articles

Extended-Interval Prophylaxis with rFIXFc in Adults and Adolescents with Hemophilia B: Interim Results of the B-YOND Extension Study

Johnny Mahlangu, Amy D Shapiro, MD, K John Pasi, Margaret V Ragni, Margareth Castro Ozelo, MD PhD, Johannes Oldenburg, Tadashi Matsushita, MD PhD, Ross I Baker, MD, Huixing Yuan, Alejandra Ramirez-Santiago, Glenn F Pierce, Geoffrey Allen, Baisong Mei

Blood (2015) 126 (23): 1088.

https://doi.org/10.1182/blood.V126.23.1088.1088

Published: 2015

Journal Articles

Low Bleeding Rates with Increase or Maintenance of Physical Activity in Patients Treated with Recombinant Factor IX Fc Fusion Protein (rFIXFc) in the B-LONG and Kids B-LONG Studies

Roshni Kulkarni, Amy D Shapiro, MD, Jerzy Windyga, Margaret V Ragni, K John Pasi, Margareth Castro Ozelo, MD PhD, Elisa Tsao, Baisong Mei

Blood (2015) 126 (23): 2307.

https://doi.org/10.1182/blood.V126.23.2307.2307

Published: 2015

Journal Articles

Long-Term Efficacy of rFVIIIFc Prophylaxis in Pediatric, Adolescent, and Adult Subjects with Target Joints and Severe Hemophilia A

Bryce A. Kerlin, Roshni Kulkarni, Beatrice Nolan, Michael Wang, MD, K John Pasi, Raina Liesner, Simon Brown, Hideji Hanabusa, Ingrid Pabinger-Fasching, Elisa Tsao, Geoffrey Allen

Blood (2015) 126 (23): 3520.

https://doi.org/10.1182/blood.V126.23.3520.3520

Published: 2015

Journal Articles

A Subcutaneously Administered Investigational RNAi Therapeutic (ALN-AT3) Targeting Antithrombin for Treatment of Hemophilia: Interim Weekly and Monthly Dosing Results in Patients with Hemophilia A or B

K John Pasi, Pencho Georgiev, Tim Mant, Michael Desmond Creagh, Toshko Lissitchkov, MD, David Bevan, Steve Austin, Charles R Hay, Brigit Brand, Rashid Kazmi, Pratima Chowdary, Margaret V Ragni, Jihong Chen, Akin Akinc, Benny Sorensen, Savita Rangarajan

Blood (2015) 126 (23): 551.

https://doi.org/10.1182/blood.V126.23.551.551

Published: 2015

Journal Articles

Personalized Prophylaxis with Human-Cl Recombinant FVIII in HA Patients

Robert Klamroth, Toshko Lissitchkov, MD, Luminita Rusen, Olaf Walter, MD MBA, Johann Bichler, PhD, K John Pasi, Andreas Tiede, MD PhD, Sigurd Knaub, PhD

Blood (2015) 126 (23): 547.

https://doi.org/10.1182/blood.V126.23.547.547

Published: 2015

NARROW

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

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

Long-Term Safety and Efficacy of rFVIIIFc in Adults and Adolescents with Severe Hemophilia A: A Longitudinal Analysis of A-LONG and ASPIRE

Extended-Interval Prophylaxis with rFIXFc in Adults and Adolescents with Hemophilia B: Interim Results of the B-YOND Extension Study

Low Bleeding Rates with Increase or Maintenance of Physical Activity in Patients Treated with Recombinant Factor IX Fc Fusion Protein (rFIXFc) in the B-LONG and Kids B-LONG Studies

Long-Term Efficacy of rFVIIIFc Prophylaxis in Pediatric, Adolescent, and Adult Subjects with Target Joints and Severe Hemophilia A

A Subcutaneously Administered Investigational RNAi Therapeutic (ALN-AT3) Targeting Antithrombin for Treatment of Hemophilia: Interim Weekly and Monthly Dosing Results in Patients with Hemophilia A or B

Personalized Prophylaxis with Human-Cl Recombinant FVIII in HA Patients

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