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Journal Articles

A Phase Ia Study of VGA039, a Protein S-Targeting Monoclonal Antibody, in Individuals with Von Willebrand Disease Demonstrates Concentration-Dependent Increases in Thrombin Generation for Reducing Bleeding

Carolyn M Millar, Priyanka Raheja, Allison P Wheeler, Shrinath Kshirsagar, Christian Schoergenhofer, Nicoletta C Machin, Cihan Ay, Almudena Tercero, Hayley Lane, Christopher J Horvath, Sandip Panicker, Gary Patou, Benjamin Kim, Savita Rangarajan

Blood (2024) 144 (Supplement 1): 3981.

https://doi.org/10.1182/blood-2024-208020

Published: 2024

Journal Articles

Factor VIII Coagulant Activity (FVIII:C) As an Independent Predictor of Bleeding across Different Subtypes of Von Willebrand Disease (VWD)

Shruti Vilas Kharat, Nayaab Khan, Alina He, Chris Foo, Kranti Patil, Gurpreetkaur Saini, Shreyas Tawde, Benjamin Kim, Savita Rangarajan, Shrimati Shetty

Blood (2024) 144 (Supplement 1): 3982.

https://doi.org/10.1182/blood-2024-209566

Published: 2024

Journal Articles

Understanding Natural History of Common Inherited Platelet Function Disorder (IPFD) Patients: An Indian Study

Shrinath Kshirsagar, Nimish Kulkarni, Shruti Kharat, Kranti Patil, Savita Rangarajan, Shrimati Shetty

Blood (2023) 142 (Supplement 1): 3966.

https://doi.org/10.1182/blood-2023-182074

Published: 2023

Journal Articles

Understanding the Disease Burden of Severe Von Willebrand Disease in a Resource Poor Country: An Indian Cohort Study

Shrinath Kshirsagar, Nimish Kulkarni, Magdalne D'silva, Savita Rangarajan, Shrimati Shetty

Blood (2023) 142 (Supplement 1): 5426.

https://doi.org/10.1182/blood-2023-179010

Published: 2023

Journal Articles

Fitusiran Reaches People's with Hemophilia and Their Caregivers’ Treatment Expectations: Qualitative Semi-Structured Interviews of Participants of ATLAS-OLE Trial (Interim analysis)

Alok Srivastava, Savita Rangarajan, Cecil Ross, Christina Slota, Dana DiBenedetti, Viridiana Cano, Shauna Andersson, Pronabesh Dasmahapatra, José Bartelt-Hofer, Marion Afonso

Blood (2022) 140 (Supplement 1): 7989–7990.

https://doi.org/10.1182/blood-2022-166895

Published: 2022

Journal Articles

Fitusiran, an Investigational siRNA Therapeutic Targeting Antithrombin for the Treatment of Hemophilia: First Results from a Phase 3 Study to Evaluate Efficacy and Safety in People with Hemophilia a or B without Inhibitors (ATLAS-A/B)

Alok Srivastava, Savita Rangarajan, Kaan Kavakli, Robert Klamroth, Gili Kenet, Liane Khoo, Chur-Woo You, Weiqun Xu, Niel Malan, Laurent Frenzel, Catherine N. Bagot, Oleksandra Stasyshyn, Chia-Yau Chang, Stacey Poloskey, Shauna Andersson, Zhiying Qiu, Baisong Mei, Steven W. Pipe

Blood (2021) 138 (Supplement 2): LBA-3.

https://doi.org/10.1182/blood-2021-155018

Published: 2021

Journal Articles

Efficacy and Safety of Fitusiran Prophylaxis, an siRNA Therapeutic, in a Multicenter Phase 3 Study (ATLAS-INH) in People with Hemophilia A or B, with Inhibitors (PwHI)

Guy Young, Alok Srivastava, Kaan Kavakli, Cecil Ross, Jameela Sathar, Huyen Tran, Runhui Wu, Jing Sun, Stacey Poloskey, Zhiying Qui, Salim Kichou, Shauna R. Andersson, Baisong Mei, Savita Rangarajan

Blood (2021) 138 (Supplement 1): 4.

https://doi.org/10.1182/blood-2021-150273

Published: 2021

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

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

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

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

Fibrinogen Concentrate Vs Cryoprecipitate in Pseudomyxoma Peritonei Surgery: Interim Results from a Prospective, Randomized, Controlled Phase 2 Study

Ashok Roy, Nigel Sargant, Savita Rangarajan, Sue Alves, John Bell, Sophia Stanford, Cristina Solomon, Irina Kruzhkova, Sigurd Knaub, Faheez Mohamed

Blood (2018) 132 (Supplement 1): 2549.

https://doi.org/10.1182/blood-2018-99-118145

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

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

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

Feiba Global Outcome Study (FEIBA-GO): Long-Term Real World Data on Apcc (Feiba R ) in Patients with Inhibitors. First Demographic Data

Ana Rosa Cid, MD, Carmen Escuriola, MD, Cedric R. Hermans, MD PhD MRCP, Pal André Holme, PhD MD, Claude Negrier, MD, Savita Rangarajan, Angiola Rocino, Jerzy Windyga, MD, Alessandro Gringeri, MD MSc, Roberto Crea, DVM, MSc

Blood (2016) 128 (22): 5038.

https://doi.org/10.1182/blood.V128.22.5038.5038

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

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

Pharmacokinetic (PK) Comparison of Two Fibrinogen Concentrates in Patients with Congenital Fibrinogen Deficiency: Final Analysis

Cecil Ross, MD, Savita Rangarajan, Mehran Karimi, MD, Bruce A. Schwartz, PhD, Sigurd Knaub, PhD, Flora Peyvandi, MD PhD

Blood (2015) 126 (23): 4680.

https://doi.org/10.1182/blood.V126.23.4680.4680

Published: 2015

NARROW

A Phase Ia Study of VGA039, a Protein S-Targeting Monoclonal Antibody, in Individuals with Von Willebrand Disease Demonstrates Concentration-Dependent Increases in Thrombin Generation for Reducing Bleeding

Factor VIII Coagulant Activity (FVIII:C) As an Independent Predictor of Bleeding across Different Subtypes of Von Willebrand Disease (VWD)

Understanding Natural History of Common Inherited Platelet Function Disorder (IPFD) Patients: An Indian Study

Understanding the Disease Burden of Severe Von Willebrand Disease in a Resource Poor Country: An Indian Cohort Study

Fitusiran Reaches People's with Hemophilia and Their Caregivers’ Treatment Expectations: Qualitative Semi-Structured Interviews of Participants of ATLAS-OLE Trial (Interim analysis)

Fitusiran, an Investigational siRNA Therapeutic Targeting Antithrombin for the Treatment of Hemophilia: First Results from a Phase 3 Study to Evaluate Efficacy and Safety in People with Hemophilia a or B without Inhibitors (ATLAS-A/B)

Efficacy and Safety of Fitusiran Prophylaxis, an siRNA Therapeutic, in a Multicenter Phase 3 Study (ATLAS-INH) in People with Hemophilia A or B, with Inhibitors (PwHI)

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

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

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

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

Fibrinogen Concentrate Vs Cryoprecipitate in Pseudomyxoma Peritonei Surgery: Interim Results from a Prospective, Randomized, Controlled Phase 2 Study

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

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

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

Feiba Global Outcome Study (FEIBA-GO): Long-Term Real World Data on Apcc (Feiba R ) in Patients with Inhibitors. First Demographic Data

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

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

Pharmacokinetic (PK) Comparison of Two Fibrinogen Concentrates in Patients with Congenital Fibrinogen Deficiency: Final Analysis

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American Society of Hematology

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American Society of Hematology

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