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Journal Articles

Long-Term Persistence of Antibodies to Adeno-Associated Viral Vectors Following Gene Therapy with scAAV8-LP1-Fixco

Jenny H. McIntosh, Saira Mohamed, Lydia Ireland, Christopher L Morton, Ulrike M. Reiss, Pratima Chowdary, Edward G. D. Tuddenham, Andrew M. Davidoff, Amit C. Nathwani

Blood (2023) 142 (Supplement 1): 2255.

https://doi.org/10.1182/blood-2023-188383

Published: 2023

Journal Articles

Design of an Observational Study (PROVE) to Assess the Long-Term Effects of Prophylaxis with Simoctocog Alfa or Emicizumab on Joint and Bone Health in Hemophilia a Patients

Robert Klamroth, Robert F Sidonio, Jr., Yesim Dargaud, Víctor Jiménez-Yuste, Rolf Ljung, Guy Young, Pratima Chowdary

Blood (2023) 142 (Supplement 1): 1255.

https://doi.org/10.1182/blood-2023-181725

Published: 2023

Journal Articles

GO-8: Stable Expression of Factor VIII over 5 Years Following Adeno-Associated Gene Transfer in Subjects with Hemophilia a Using a Novel Human Factor VIII Variant

Blood (2023) 142 (Supplement 1): 3624.

https://doi.org/10.1182/blood-2023-180803

Published: 2023

Journal Articles

Stable Therapeutic Transgenic FIX Levels for More Than 10 Years in Subjects with Severe Hemophilia B Who Received scAAV2/8-LP1-Hfixco Adeno-Associated Virus Gene Therapy

Blood (2023) 142 (Supplement 1): 1056.

https://doi.org/10.1182/blood-2023-186891

Published: 2023

Journal Articles

A Multiple Ascending Dose Study of Mim8 in Patients with Severe Hemophilia a with or without Factor VIII Inhibitors

Steven R. Lentz, Pratima Chowdary, Francisco Jose Lopez Jaime, Johnny Mahlangu, Irina Matytsina, Anne Louise Nielsen, Jerzy Windyga

Blood (2022) 140 (Supplement 1): 2735–2736.

https://doi.org/10.1182/blood-2022-160043

Published: 2022

Journal Articles

Efficacy of Efanesoctocog Alfa on Pain in Patients with Hemophilia A: Results from the XTEND-1 Phase 3 Clinical Trial in Previously Treated Patients with Hemophilia A

Amanda Wilson, Nana Kragh, Jerome Mshijid, Jennifer Dumont, Annemieke Willemze, Elena Santagostino, Chuanwu Zhang, Roshni Kulkarni, Annette von Drygalski, Pratima Chowdary

Blood (2022) 140 (Supplement 1): 5610–5611.

https://doi.org/10.1182/blood-2022-166849

Published: 2022

Journal Articles

Prophylactic Treatment with Rurioctocog Alfa Pegol Results in a Dose-Dependent Normalization of Biomarkers of Joint Health in Severe Hemophilia a: An Exploratory Analysis from the Propel Study

Tina Manon-Jensen, Srilatha Tangada, Cecilie Bager, Pratima Chowdary, Robert Klamroth, Annette von Drygalski, Jerzy Windyga, Miguel A. Escobar, Peder Frederiksen, Werner Engl, Bruce Ewenstein, Morten Karsdal

Blood (2021) 138 (Supplement 1): 2102.

https://doi.org/10.1182/blood-2021-150139

Published: 2021

Journal Articles

Emicizumab Prophylaxis in Persons with Mild or Moderate Hemophilia A: Results from the Interim Analysis of the HAVEN 6 Study

Claude Négrier, Johnny Mahlangu, Michaela Lehle, Pratima Chowdary, Olivier Catalani, Víctor Jiménez-Yuste, Benjamin M. Beckermann, Christophe Schmitt, Cedric Hermans, Giuliana Ventriglia, Jerzy Windyga, Anna Kiialainen, Roseline d'Oiron, Paul Moorehead, Vanda Teodoro, Amy D. Shapiro, Johannes Oldenburg

Blood (2021) 138 (Supplement 1): 343.

https://doi.org/10.1182/blood-2021-146009

Published: 2021

Journal Articles

Factor IX Expression within the Normal Range Prevents Spontaneous Bleeds Requiring Treatment Following FLT180a Gene Therapy in Patients with Severe Hemophilia B: Long-Term Follow-up Study of the B-Amaze Program

Pratima Chowdary, Susan Shapiro, Mike Makris, Gillian Evans, Sara Boyce, Kate Talks, Gerry Dolan, Ulrike Reiss, Mark Phillips, Anne Riddell, Maria Rita Peralta, Michelle Quaye, Ted Tuddenham, Alison Long, Julie Krop, Amit Nathwani

Blood (2021) 138 (Supplement 1): 3967.

https://doi.org/10.1182/blood-2021-146546

Published: 2021

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

Fitusiran Treatment Impacts on Health-Related Quality of Life in Subjects with Hemophilia A and B with Inhibitors Assessed with the Haemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL)

Sylvia Von Mackensen, Pratima Chowdary, Sarah Mangles, Qifeng Yu, Baisong Mei, Shauna R. Andersson, Pronabesh Dasmahapatra, MBBS, MPH

Blood (2020) 136 (Supplement 1): 23–24.

https://doi.org/10.1182/blood-2020-142266

Published: 2020

Journal Articles

Subcutaneous concizumab prophylaxis in hemophilia A and hemophilia A/B with inhibitors: phase 2 trial results

Amy D. Shapiro, Pantep Angchaisuksiri, Jan Astermark, Gary Benson, Giancarlo Castaman, Pratima Chowdary, Hermann Eichler, Victor Jiménez-Yuste, Kaan Kavakli, Tadashi Matsushita, Lone Hvitfeldt Poulsen, Allison P. Wheeler, Guy Young, Silva Zupancic-Salek, Johannes Oldenburg

Blood (2019) 134 (22): 1973–1982.

https://doi.org/10.1182/blood.2019001542

Published: 2019

Includes: Multimedia, Supplemental data

Journal Articles

Efficacy and Safety of Subcutaneous Prophylaxis with Concizumab in Patients with Severe Hemophilia a without Inhibitors: Results from the Phase 2 explorer5 Trial

Hermann Eichler, Pantep Angchaisuksiri, MD, Jan Astermark, MD PhD, Gary Benson, Katarina Cepo, Pratima Chowdary, MD, Ute Friedrich, PhD, Kaan Kavakli, Allison P Wheeler, MD, Johannes Oldenburg, MD PhD

Blood (2019) 134 (Supplement_1): 2417.

https://doi.org/10.1182/blood-2019-122300

Published: 2019

Journal Articles

A Single Intravenous Infusion of FLT180a Results in Factor IX Activity Levels of More Than 40% and Has the Potential to Provide a Functional Cure for Patients with Haemophilia B

Pratima Chowdary, MD, Susie Shapiro, MD, Andrew M. Davidoff, MD, Ulrike Reiss, MD, Rebecca Alade, PhD, Geoff Brooks, BSc, Allison Dane, PhD, Jenny McIntosh, PhD, Gerard Short, MBBS, Edward Tuddenham, MD, Amit C Nathwani, MD PhD

Blood (2018) 132 (Supplement 1): 631.

https://doi.org/10.1182/blood-2018-99-118050

Published: 2018

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

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

GO-8: Preliminary Results of a Phase I/II Dose Escalation Trial of Gene Therapy for Haemophilia a Using a Novel Human Factor VIII Variant

Amit C Nathwani, MD PhD, Edward Tuddenham, MD, Pratima Chowdary, MD, Jenny McIntosh, PhD, Doyoung Lee, PhD, Cecilia Rosales, PhD, Mark Phillips, Jun Pie, Zhou Junfang, Michael M Meagher, PhD, Ulrike Reiss, MD, Andrew M. Davidoff, MD, Chris L Morton, Anne Riddell

Blood (2018) 132 (Supplement 1): 489.

https://doi.org/10.1182/blood-2018-99-118256

Published: 2018

Journal Articles

Pharmacodynamics, Pharmacokinetics and Safety of Bay 1093884, an Antibody Directed Against Human TFPI, in Patients with Factor VIII or IX Deficiency (With and Without Inhibitors): A Phase 1 Study

Pratima Chowdary, MD, Toshko Jelev Lissitchkov, MD, Stefan Willmann, Dr, Stephan Schwers, PhD, Lisa A. Michaels, Anita Shah

Blood (2018) 132 (Supplement 1): 1176.

https://doi.org/10.1182/blood-2018-99-114683

Published: 2018

Journal Articles

Efficacy of an Extended Half-Life GlycoPEGylated rFVIII (N8-GP): Pooled Analysis of ABR (Results from Two Clinical Trials)

Pratima Chowdary, MD, Lone Hvitfeldt Poulsen, Miguel A Escobar, MD, Susan Kearney, Meera B. Chitlur, MD, Robert Klamroth, Mudi Misgav, Claude Negrier, Wan Hui Ong Clausen, Frank Driessler, Andrea Landorph, Elena Santagostino

Blood (2018) 132 (Supplement 1): 1177.

https://doi.org/10.1182/blood-2018-99-110308

Published: 2018

Journal Articles

Concizumab (Anti-TFPI) Exposure-Response Modeling in Patients with Hemophilia A

Hermann Eichler, MD, Pantep Angchaisuksiri, MD, Kaan Kavakli, MD, Paul Knöbl, MD PhD, Jerzy Windyga, Victor Jimenez-Yuste, Ute Friedrich, PhD, Philip Delff Andersen, Pratima Chowdary

Blood (2017) 130 (Supplement 1): 3672.

https://doi.org/10.1182/blood.V130.Suppl_1.3672.3672

Published: 2017

NARROW

Long-Term Persistence of Antibodies to Adeno-Associated Viral Vectors Following Gene Therapy with scAAV8-LP1-Fixco

Design of an Observational Study (PROVE) to Assess the Long-Term Effects of Prophylaxis with Simoctocog Alfa or Emicizumab on Joint and Bone Health in Hemophilia a Patients

GO-8: Stable Expression of Factor VIII over 5 Years Following Adeno-Associated Gene Transfer in Subjects with Hemophilia a Using a Novel Human Factor VIII Variant

Stable Therapeutic Transgenic FIX Levels for More Than 10 Years in Subjects with Severe Hemophilia B Who Received scAAV2/8-LP1-Hfixco Adeno-Associated Virus Gene Therapy

A Multiple Ascending Dose Study of Mim8 in Patients with Severe Hemophilia a with or without Factor VIII Inhibitors

Efficacy of Efanesoctocog Alfa on Pain in Patients with Hemophilia A: Results from the XTEND-1 Phase 3 Clinical Trial in Previously Treated Patients with Hemophilia A

Prophylactic Treatment with Rurioctocog Alfa Pegol Results in a Dose-Dependent Normalization of Biomarkers of Joint Health in Severe Hemophilia a: An Exploratory Analysis from the Propel Study

Emicizumab Prophylaxis in Persons with Mild or Moderate Hemophilia A: Results from the Interim Analysis of the HAVEN 6 Study

Factor IX Expression within the Normal Range Prevents Spontaneous Bleeds Requiring Treatment Following FLT180a Gene Therapy in Patients with Severe Hemophilia B: Long-Term Follow-up Study of the B-Amaze Program

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

Fitusiran Treatment Impacts on Health-Related Quality of Life in Subjects with Hemophilia A and B with Inhibitors Assessed with the Haemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL)

Subcutaneous concizumab prophylaxis in hemophilia A and hemophilia A/B with inhibitors: phase 2 trial results

Efficacy and Safety of Subcutaneous Prophylaxis with Concizumab in Patients with Severe Hemophilia a without Inhibitors: Results from the Phase 2 explorer5 Trial

A Single Intravenous Infusion of FLT180a Results in Factor IX Activity Levels of More Than 40% and Has the Potential to Provide a Functional Cure for Patients with Haemophilia B

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

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

GO-8: Preliminary Results of a Phase I/II Dose Escalation Trial of Gene Therapy for Haemophilia a Using a Novel Human Factor VIII Variant

Pharmacodynamics, Pharmacokinetics and Safety of Bay 1093884, an Antibody Directed Against Human TFPI, in Patients with Factor VIII or IX Deficiency (With and Without Inhibitors): A Phase 1 Study

Efficacy of an Extended Half-Life GlycoPEGylated rFVIII (N8-GP): Pooled Analysis of ABR (Results from Two Clinical Trials)

Concizumab (Anti-TFPI) Exposure-Response Modeling in Patients with Hemophilia A

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American Society of Hematology

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American Society of Hematology

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