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Journal Articles

Concizumab efficacy in patients with Hemophilia A/B without inhibitors from the Phase 3 explorer8 study: A post-hoc sensitivity analysis for the intra-patient comparison of concizumab with previous prophylaxis

Anthony Chan, Hermann Eichler, Julien Bovet, Renée Brown Frandsen, Allison Wheeler, Pratima Chowdary

Blood (2025) 146 (Supplement 1): 1288.

https://doi.org/10.1182/blood-2025-1288

Published: 2025

Journal Articles

Clinical outcomes up to 4 years of once-weekly efanesoctocog alfa prophylaxis in previously treated adults, adolescents, and children with severe hemophilia A: Interim analysis of the Phase 3 XTEND-ed long-term extension study

Sophie Susen, Anthony Chan, Pratima Chowdary, Stephanie P'ng, Manuela Albisetti, Keiji Nogami, Karin Fijnvandraat, Jennifer Dumont, Elena Santagostino, Sriya Gunawardena, Mahnouch Georget, Lynn Malec

Blood (2025) 146 (Supplement 1): 539.

https://doi.org/10.1182/blood-2025-539

Published: 2025

Journal Articles

Mim8 prophylaxis in adults and adolescents with hemophilia A: 52-week efficacy and safety outcomes from the phase 3 FRONTIER2 study

Steven Lentz, Aby Abraham, Cihan Ay, Anthony Chan, Víctor Jiménez-Yuste, Johannes Oldenburg, Maria Elisa Mancuso, Johnny Mahlangu, Tadashi Matsushita, Lize van Vulpen, Renchi Yang, Amalie Rhode Høgh Nielsen, Ilgiz Rakhmatullin, Pratima Chowdary

Blood (2025) 146 (Supplement 1): 537.

https://doi.org/10.1182/blood-2025-537

Published: 2025

Journal Articles

Adeno-associated viral integration profiles in human liver biopsies following Hemophilia A gene therapy

Paul Batty, Samuel Markey, Jenny McIntosh, Giulia Simini, Edward Tuddenham, Pratima Chowdary, Amit Nathwani

Blood (2025) 146 (Supplement 1): 4315.

https://doi.org/10.1182/blood-2025-4315

Published: 2025

Journal Articles

Decoding Transgene Silencing from Stably Maintained AAV Genomes in Human Liver: Epigenetic Insights to Guide Next-Generation Vector Design

Giulia Simini, Jenny McIntosh, Vincent Muczynski, Malo Journou, Paul Batty, Pratima Chowdary, Christopher Morton, Andrew Davidoff, Amit Nathwani

Blood (2025) 146 (Supplement 1): 4310.

https://doi.org/10.1182/blood-2025-4310

Published: 2025

Journal Articles

Patient and Caregiver Preferences for Hemophilia Prophylactic Treatments: A Discrete Choice Experiment

Hui Lu, Jenny Whitty, Travis Gould, Carmine Colavecchia, Josh Coulter, Nicola Dunn, Pratima Chowdary, Lisa J Wilcox, Joseph C. Cappelleri, Stephanie A Christopher, Alexis Sohn, Brett Hauber

Blood (2024) 144 (Supplement 1): 2591.

https://doi.org/10.1182/blood-2024-207425

Published: 2024

Journal Articles

Safety and Efficacy of Mim8 Prophylaxis Administered Once Every Two Weeks for Patients with Hemophilia A with or without Inhibitors: Interim Analysis of the FRONTIER4 Open-Label Extension Study

Tadashi Matsushita, Pratima Chowdary, Atanas Mitkov Banchev, Kaan Kavakli, Johanna A. Kremer Hovinga, Jerzy Windyga, Víctor Jiménez-Yuste, Julien Bovet, Llenalia María García Fernández, Guy Young

Blood (2024) 144 (Supplement 1): 718.

https://doi.org/10.1182/blood-2024-198617

Published: 2024

Journal Articles

Clinical Outcomes over 3 Years of Once-Weekly Efanesoctocog Alfa Treatment in Adults and Adolescents with Severe Hemophilia A: Second Interim Analysis from the Phase 3 XTEND-ed Long-Term Extension Study

Robert Klamroth, Stephanie P'ng, Sophie Susen, Davide Matino, Pratima Chowdary, Angela C Weyand, Toshko Lissitchkov, Young-Shil Park, María Teresa Alvarez-Román, Liqi Feng, Helena Palmborg, Jennifer Dumont, Elena Santagostino, Lara Mamikonian, Keiji Nogami

Blood (2024) 144 (Supplement 1): 717.

https://doi.org/10.1182/blood-2024-204533

Published: 2024

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

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

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

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

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

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

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

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

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

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

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

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Concizumab efficacy in patients with Hemophilia A/B without inhibitors from the Phase 3 explorer8 study: A post-hoc sensitivity analysis for the intra-patient comparison of concizumab with previous prophylaxis

Clinical outcomes up to 4 years of once-weekly efanesoctocog alfa prophylaxis in previously treated adults, adolescents, and children with severe hemophilia A: Interim analysis of the Phase 3 XTEND-ed long-term extension study

Mim8 prophylaxis in adults and adolescents with hemophilia A: 52-week efficacy and safety outcomes from the phase 3 FRONTIER2 study

Adeno-associated viral integration profiles in human liver biopsies following Hemophilia A gene therapy

Decoding Transgene Silencing from Stably Maintained AAV Genomes in Human Liver: Epigenetic Insights to Guide Next-Generation Vector Design

Patient and Caregiver Preferences for Hemophilia Prophylactic Treatments: A Discrete Choice Experiment

Safety and Efficacy of Mim8 Prophylaxis Administered Once Every Two Weeks for Patients with Hemophilia A with or without Inhibitors: Interim Analysis of the FRONTIER4 Open-Label Extension Study

Clinical Outcomes over 3 Years of Once-Weekly Efanesoctocog Alfa Treatment in Adults and Adolescents with Severe Hemophilia A: Second Interim Analysis from the Phase 3 XTEND-ed Long-Term Extension Study

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

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

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

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

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

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

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

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

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)

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

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

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