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Journal Articles

Bleeding Outcomes in Participants with Factor VIII Activity <5 IU/DL Post-Gene Transfer in GENEr8-1

Johnny Mahlangu, Margareth Castro Ozelo, Chee Wee Tan, Adam Giermasz, Jiaan-Der Wang, Dane Osmond, Hua Yu, Tara M. Robinson

Blood (2023) 142 (Supplement 1): 2250.

https://doi.org/10.1182/blood-2023-180083

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

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

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

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

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

Journal Articles

Eptacog Beta (rFVIIa) Has a Low Incidence of Spontaneous Rebleeding through 24 and 48 Hours in Adult and Adolescent Patients with Hemophilia A or B with Inhibitors

Amy L. Dunn, Yesim Dargaud, Yasmina L. Abajas, Manuel Carcao, Giancarlo Castaman, Adam Giermasz, Cédric Hermans, Magdalena D. Lewandowska, Johnny Mahlangu, Shannon L. Meeks, Wolfgang A. Miesbach, Michael Recht, Vanessa Salinas, Tammuella Chrisentery-Singleton, Hongying Wang, Ian S Mitchell, Guy Young

Blood (2022) 140 (Supplement 1): 8485–8488.

https://doi.org/10.1182/blood-2022-157343

Published: 2022

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

A randomized, placebo-controlled, double-blind trial of canakinumab in children and young adults with sickle cell anemia

Blood (2022) 139 (17): 2642–2652.

https://doi.org/10.1182/blood.2021013674

Published: 2022

Includes: Supplemental data

Journal Articles

Health-Related Quality of Life Following Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A in the Phase 3 Trial GENEr8-1

Blood (2021) 138 (Supplement 1): 4916.

https://doi.org/10.1182/blood-2021-146021

Published: 2021

Journal Articles

Eptacog Beta Efficacy in Children and Adolescents with Hemophilia A or B and Inhibitors: Subset Analysis Suggests Improved Caregiver Capacity to Assess Bleeding Episode Resolution with Subject Age

Blood (2021) 138 (Supplement 1): 3195.

https://doi.org/10.1182/blood-2021-147434

Published: 2021

Journal Articles

Emicizumab Prophylaxis in Persons with Hemophilia A, Aged ≥50 Years, with Comorbidities - Pooled Data from Four Phase III Studies (HAVEN 1, 3, and 4, and STASEY)

Víctor Jiménez-Yuste, Johannes Oldenburg, Eunice Tzeng, Ben Trzaskoma, Fabian Sanabria, Johnny Mahlangu

Blood (2021) 138 (Supplement 1): 2103.

https://doi.org/10.1182/blood-2021-145703

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

Long-term outcomes with emicizumab prophylaxis for hemophilia A with or without FVIII inhibitors from the HAVEN 1-4 studies

Blood (2021) 137 (16): 2231–2242.

https://doi.org/10.1182/blood.2020009217

Published: 2021

Includes: Supplemental data

Journal Articles

Safety and Efficacy of Emicizumab in Persons with Hemophilia a with or without FVIII Inhibitors: Pooled Data from Four Phase III Studies (HAVEN 1-4)

Blood (2020) 136 (Supplement 1): 3–5.

https://doi.org/10.1182/blood-2020-137438

Published: 2020

Journal Articles

Immunoglobulin Sub-Typing and Quantification in Coombs Positive Hemolysis

Thirosha Chetty, MD, Nikki Bouwer, MD, Yuen On Wan, MD, Johnny Mahlangu

Blood (2020) 136 (Supplement 1): 19–20.

https://doi.org/10.1182/blood-2020-138452

Published: 2020

Journal Articles

A multicenter, open-label phase 3 study of emicizumab prophylaxis in children with hemophilia A with inhibitors

Guy Young, Ri Liesner, Tiffany Chang, Robert Sidonio, Jr, Johannes Oldenburg, Victor Jiménez-Yuste, Johnny Mahlangu, Rebecca Kruse-Jarres, Michael Wang, Marianne Uguen, Michelle Y. Doral, Lilyan Y. Wright, Christophe Schmitt, Gallia G. Levy, Midori Shima, Maria Elisa Mancuso

Blood (2019) 134 (24): 2127–2138.

https://doi.org/10.1182/blood.2019001869

Published: 2019

Includes: Supplemental data

Journal Articles

Interim Analysis of the Extension Study with rVIII-SingleChain in Previously Untreated Patients (PUPs) with Severe Hemophilia A (CSL627-3001)

Elena Santagostino, Kathelijn Fischer, Christoph Koenigs, MD PhD, Claudia Djambas Khayat, MD BSc, Samantha Lucas, Blanca Salazar, Andres Brainsky, MD, Thomas Chung, Brahm Goldstein, Johnny Mahlangu, MMed, FCPath

Blood (2019) 134 (Supplement_1): 162.

https://doi.org/10.1182/blood-2019-123981

Published: 2019

Journal Articles

Factor VIII Use in the Treatment of Breakthrough Bleeds in Hemophilia A Patients without Inhibitors on Emicizumab Prophylaxis: The Phase 3 HAVEN 3 Study Experience

Michael Callaghan, MD, Benjamin Trzaskoma, MS, Richard H. Ko, MD, Lucy Lee, PharmD, Anisha M. Patel, PhD, Eunice Tzeng, PharmD, Michelle Lynn Shah, PharmD, Tiffany Y. Chang, MD MAS, Markus Niggli, PhD, Christophe Dhalluin, PhD, Johnny Mahlangu, MMed, FCPath

Blood (2019) 134 (Supplement_1): 2395.

https://doi.org/10.1182/blood-2019-123654

Published: 2019

NARROW

Bleeding Outcomes in Participants with Factor VIII Activity <5 IU/DL Post-Gene Transfer in GENEr8-1

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

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

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

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

Eptacog Beta (rFVIIa) Has a Low Incidence of Spontaneous Rebleeding through 24 and 48 Hours in Adult and Adolescent Patients with Hemophilia A or B with Inhibitors

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

A randomized, placebo-controlled, double-blind trial of canakinumab in children and young adults with sickle cell anemia

Health-Related Quality of Life Following Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A in the Phase 3 Trial GENEr8-1

Eptacog Beta Efficacy in Children and Adolescents with Hemophilia A or B and Inhibitors: Subset Analysis Suggests Improved Caregiver Capacity to Assess Bleeding Episode Resolution with Subject Age

Emicizumab Prophylaxis in Persons with Hemophilia A, Aged ≥50 Years, with Comorbidities - Pooled Data from Four Phase III Studies (HAVEN 1, 3, and 4, and STASEY)

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

Long-term outcomes with emicizumab prophylaxis for hemophilia A with or without FVIII inhibitors from the HAVEN 1-4 studies

Safety and Efficacy of Emicizumab in Persons with Hemophilia a with or without FVIII Inhibitors: Pooled Data from Four Phase III Studies (HAVEN 1-4)

Immunoglobulin Sub-Typing and Quantification in Coombs Positive Hemolysis

A multicenter, open-label phase 3 study of emicizumab prophylaxis in children with hemophilia A with inhibitors

Interim Analysis of the Extension Study with rVIII-SingleChain in Previously Untreated Patients (PUPs) with Severe Hemophilia A (CSL627-3001)

Factor VIII Use in the Treatment of Breakthrough Bleeds in Hemophilia A Patients without Inhibitors on Emicizumab Prophylaxis: The Phase 3 HAVEN 3 Study Experience

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American Society of Hematology

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ASH Publications

American Society of Hematology

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