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Journal Articles

Efficacy and Safety of Marstacimab Prophylaxis in Hemophilia A/B With Inhibitors: Results from the Phase 3 BASIS Trial

Davide Matino, Suchitra Acharya, Carrie Turich Taylor, Pengling Sun, Delphine Agathon, Sangeeta Raje, Travis Gould, Andrew Palladino, Johnny Mahlangu

Blood blood.2025031065.

https://doi.org/10.1182/blood.2025031065

Published: 2025

Includes: Supplemental data

Journal Articles

Bridging the gap: Global caregiver perspectives on awareness, motivators, and barriers to clinical trial participation in sickle cell disease

John James, Johnny Mahlangu, Raffaella Colombatti, Cassandra Trimnell, Darren Fleetwood, Emma Tan, Biree Andemariam

Blood (2025) 146 (Supplement 1): 2952.

https://doi.org/10.1182/blood-2025-2952

Published: 2025

Journal Articles

Pociredir, a novel oral once-daily fetal hemoglobin inducer: Results from the Phase 1b pioneer study in adult participants with severe sickle cell disease and hydroxyurea intolerance or unresponsiveness

Sheinei Alan, Johnny Mahlangu, Michael Birrer, Marshall Stagg, Debra Ferman, Rita Lemming, Caterina Minniti, Thomas Winkler, Adeyemi Adenola, Modupe Idowu

Blood (2025) 146 (Supplement 1): 1157.

https://doi.org/10.1182/blood-2025-1157

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

Trust and relationships between people with sickle cell disease and healthcare professionals: Regional comparisons from the listen survey

Biree Andemariam, Shashidhar Rao, Rohan Kolla, Emma Tan, Johnny Mahlangu

Blood (2025) 146 (Supplement 1): 2669.

https://doi.org/10.1182/blood-2025-2669

Published: 2025

Journal Articles

Long term effect of marstacimab prophylaxis in hemophilia Α and Β on target joints: Results from BASIS and OLE studies

Víctor Jiménez-Yuste, Johnny Mahlangu, Hae Kyung Kim, Travis Gould, Eugeny Mefyod, Pengling Sun, Bryan McComb, Carrie Turich Taylor, Andrew Palladino

Blood (2025) 146 (Supplement 1): 4844.

https://doi.org/10.1182/blood-2025-4844

Published: 2025

Journal Articles

Concizumab plasma concentration measurements for personalized dose adjustment in patients with Hemophilia A/B with and without inhibitors: Data from the Phase 3 explorer7 and explorer8 studies

Hermann Eichler, Chris Barnes, Johnny Mahlangu, Mary Mathias, Jakob Oemar Gjørret, Thomas Porstmann, Tulika Seth, Sonata Šaulytė Trakymienė

Blood (2025) 146 (Supplement 1): 3070.

https://doi.org/10.1182/blood-2025-3070

Published: 2025

Journal Articles

Subcutaneous, every-four-week maintenance dosing of a novel protein S antibody is well-tolerated and substantially reduces bleeding rates: Results from A phase 1/2 multidose study of VGA039 in patients with von Willebrand disease

Allison Wheeler, Shrinath Kshirsagar, Adam Giermasz, Johnny Mahlangu, Gabriela Yamaguti-Hayakawa, Savita Rangarajan, Genevieve Moyer, Christine Kempton, Almudena Tercero, Paul Sims, Sandip Panicker, Chris Foo, Eileen Sawyer, Chris Del Nagro, Gary Patou, Benjamin Kim, Nicoletta Machin

Blood (2025) 146 (Supplement 1): 308.

https://doi.org/10.1182/blood-2025-308

Published: 2025

Journal Articles

Marstacimab prophylaxis in participants with Hemophilia A or B with inhibitors: Results from the Phase 3 BASIS trial

Davide Matino, Suchitra Acharya, Carrie Turich Taylor, Pengling Sun, Delphine Agathon, Travis Gould, Andrew Palladino, Johnny Mahlangu

Blood (2025) 146 (Supplement 1): 306.

https://doi.org/10.1182/blood-2025-306

Published: 2025

Journal Articles

Marstacimab prophylaxis in hemophilia A/B without inhibitors: results from the phase 3 BASIS trial

Davide Matino, Andrew Palladino, Carrie Turich Taylor, Eunhee Hwang, Sangeeta Raje, Satyaprakash Nayak, Regina McDonald, Suchitra S. Acharya, Johnny Mahlangu, Victor Jiménez-Yuste, Nirmalkumar Choraria, Renchi Yang, Chi-kong Li, Murtadha Al-Khabori, Yasser Wali, Javier Morales Adrián, Young-Shil Park, O. Bülent Zülfikar, John Teeter

Blood (2025) 146 (14): 1654–1663.

https://doi.org/10.1182/blood.2024027468

Published: 2025

Includes: Supplemental data

Journal Articles

How People with Sickle Cell Disease Rate Motivators is Associated with the Likelihood of Wanting to Participate in a Clinical Trial: Findings from the Global LISTEN Survey

Biree Andemariam, Johnny Mahlangu, Raffaella Colombatti, John Waller, Samar Al-Behaisi, Gareth Morrell, John James

Blood (2024) 144 (Supplement 1): 1137.

https://doi.org/10.1182/blood-2024-200009

Published: 2024

Journal Articles

Pain Reduction Following Treatment of Bleeding Episodes with Eptacog Beta in Adults and Adolescents with Hemophilia A or B Complicated By Inhibitors

Tyler W. Buckner, Craig Kessler, Giancarlo Castaman, Cédric Hermans, Victor Jimenez-Yuste, Johnny Mahlangu, Wolfgang A. Miesbach, Johannes Oldenburg, Michael Recht, Leonard A. Valentino, Allison P Wheeler, Steven W. Pipe

Blood (2024) 144 (Supplement 1): 3972.

https://doi.org/10.1182/blood-2024-194935

Published: 2024

Journal Articles

Annualized Bleeding Rates in Patients with Hemophilia a or B and Inhibitors with and without Target Joints at Baseline: Results from the Concizumab Phase 3 Explorer7 Study

Amy D. Shapiro, Shashikant Apte, Ana Boban, Renée Brown Frandsen, Silvia Linari, Johnny Mahlangu, Camila Martins Mazini Tavares, Tadashi Matsushita, Salim Nekkal, Jameela Sathar, Anthony K C Chan

Blood (2024) 144 (Supplement 1): 715.

https://doi.org/10.1182/blood-2024-209759

Published: 2024

Journal Articles

Characterizing People with Sickle Cell Disease Who Share Attitudes Regarding Clinical Trial Participation: Findings from the Global LISTEN Survey

Johnny Mahlangu, John James, Biree Andemariam, John Waller, Samar Al-Behaisi, Gareth Morrell, Raffaella Colombatti, Cassandra Trimnell

Blood (2024) 144 (Supplement 1): 1135.

https://doi.org/10.1182/blood-2024-200550

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

Motivators and Barriers for People with Sickle Cell Disease Participating in Clinical Trials: Global Findings from the LISTEN Survey

John James, Biree Andemariam, Johnny Mahlangu, Raffaella Colombatti, John Waller, Samar Al-Behaisi, Adam Wufsus, Gareth Morrell, Cassandra Trimnell

Blood (2023) 142 (Supplement 1): 2498.

https://doi.org/10.1182/blood-2023-181910

Published: 2023

Journal Articles

Low Rebleed Incidence with Eptacog Beta Treatment through 24 and 48 Hours in Children (<12 years) with Hemophilia a or B with Inhibitors

Blood (2023) 142 (Supplement 1): 5480.

https://doi.org/10.1182/blood-2023-181299

Published: 2023

Journal Articles

Trials in Progress: The Randomized, Double-Blind, Placebo-Controlled Phase 1b CROSSWALK-a and Phase 2a CROSSWALK-c Trials - Crovalimab for the Treatment and Prevention of Vaso-Occlusive Episodes in Sickle Cell Disease

Marco Aurelio Salvino, Bulent Antmen, Frederick Chite Asirwa, Satheesh Chonat, Lucia De Franceschi, Perla Eleftheriou, Adlette Inati, Johnny Mahlangu, Erfan Nur, Salvador Payán-Pernía, Asad Charania, Diana Gradinaru, Allison Hsia, Yves Luder, Thomas Perretti, Sasha Srekovic, Pablo Bartolucci

Blood (2023) 142 (Supplement 1): 1158.

https://doi.org/10.1182/blood-2023-179858

Published: 2023

Journal Articles

Emicizumab Prophylaxis in People with Moderate or Mild Hemophilia A Aged ≥40 Years, with and without Comorbidities - Findings from the Phase III HAVEN 6 Study

Víctor Jiménez-Yuste, Eunice Tzeng, Elise Lim, Giuliana Ventriglia, Amy D. Shapiro, Johannes Oldenburg, Johnny Mahlangu

Blood (2023) 142 (Supplement 1): 5495.

https://doi.org/10.1182/blood-2023-173016

Published: 2023

Journal Articles

Marstacimab, an Anti-Tissue Factor Pathway Inhibitor, in Participants with Hemophilia Α or B, with and without Inhibitors: An Integrated Analysis of Safety

Suchitra Acharya, Davide Matino, Johnny Mahlangu, Carrie Turich Taylor, Eunhee Hwang, Tina Hinnershitz, Eugeny Mefyod, Andrew Palladino, Francesca Biondo, Travis Gould, John Teeter

Blood (2023) 142 (Supplement 1): 3980.

https://doi.org/10.1182/blood-2023-174682

Published: 2023

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Efficacy and Safety of Marstacimab Prophylaxis in Hemophilia A/B With Inhibitors: Results from the Phase 3 BASIS Trial

Bridging the gap: Global caregiver perspectives on awareness, motivators, and barriers to clinical trial participation in sickle cell disease

Pociredir, a novel oral once-daily fetal hemoglobin inducer: Results from the Phase 1b pioneer study in adult participants with severe sickle cell disease and hydroxyurea intolerance or unresponsiveness

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

Trust and relationships between people with sickle cell disease and healthcare professionals: Regional comparisons from the listen survey

Long term effect of marstacimab prophylaxis in hemophilia Α and Β on target joints: Results from BASIS and OLE studies

Concizumab plasma concentration measurements for personalized dose adjustment in patients with Hemophilia A/B with and without inhibitors: Data from the Phase 3 explorer7 and explorer8 studies

Subcutaneous, every-four-week maintenance dosing of a novel protein S antibody is well-tolerated and substantially reduces bleeding rates: Results from A phase 1/2 multidose study of VGA039 in patients with von Willebrand disease

Marstacimab prophylaxis in participants with Hemophilia A or B with inhibitors: Results from the Phase 3 BASIS trial

Marstacimab prophylaxis in hemophilia A/B without inhibitors: results from the phase 3 BASIS trial

How People with Sickle Cell Disease Rate Motivators is Associated with the Likelihood of Wanting to Participate in a Clinical Trial: Findings from the Global LISTEN Survey

Pain Reduction Following Treatment of Bleeding Episodes with Eptacog Beta in Adults and Adolescents with Hemophilia A or B Complicated By Inhibitors

Annualized Bleeding Rates in Patients with Hemophilia a or B and Inhibitors with and without Target Joints at Baseline: Results from the Concizumab Phase 3 Explorer7 Study

Characterizing People with Sickle Cell Disease Who Share Attitudes Regarding Clinical Trial Participation: Findings from the Global LISTEN Survey

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

Motivators and Barriers for People with Sickle Cell Disease Participating in Clinical Trials: Global Findings from the LISTEN Survey

Low Rebleed Incidence with Eptacog Beta Treatment through 24 and 48 Hours in Children (<12 years) with Hemophilia a or B with Inhibitors

Trials in Progress: The Randomized, Double-Blind, Placebo-Controlled Phase 1b CROSSWALK-a and Phase 2a CROSSWALK-c Trials - Crovalimab for the Treatment and Prevention of Vaso-Occlusive Episodes in Sickle Cell Disease

Emicizumab Prophylaxis in People with Moderate or Mild Hemophilia A Aged ≥40 Years, with and without Comorbidities - Findings from the Phase III HAVEN 6 Study

Marstacimab, an Anti-Tissue Factor Pathway Inhibitor, in Participants with Hemophilia Α or B, with and without Inhibitors: An Integrated Analysis of Safety

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American Society of Hematology

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