Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A
Ozelo, Margareth C, Mahlangu, Johnny, Pasi, K. John, Giermasz, Adam, Leavitt, Andrew D, Laffan, Michael, Symington, Emily, Quon, Doris V, Wang, Jiaan-Der, Peerlinck, Kathelijne, Pipe, Steven W, Madan, Bella, Key, Nigel S, Pierce, Glenn F, O’Mahony, Brian, Kaczmarek, Radoslaw, Henshaw, Joshua, Lawal, Adebayo, Jayaram, Kala, Huang, Mei, Yang, Xinqun, Wong, Wing Y, Kim, Benjamin
Published in The New England journal of medicine (17.03.2022)
Published in The New England journal of medicine (17.03.2022)
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Journal Article
Bleeding Outcomes in Participants with Factor VIII Activity <5 IU/DL Post-Gene Transfer in GENEr8-1
Mahlangu, Johnny, Ozelo, Margareth Castro, Tan, Chee Wee, Giermasz, Adam, Wang, Jiaan-Der, Osmond, Dane, Yu, Hua, Robinson, Tara M.
Published in Blood (02.11.2023)
Published in Blood (02.11.2023)
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Journal Article
Motivators and Barriers for People with Sickle Cell Disease Participating in Clinical Trials: Global Findings from the LISTEN Survey
James, John, Andemariam, Biree, Mahlangu, Johnny, Colombatti, Raffaella, Waller, John, Al-Behaisi, Samar, Wufsus, Adam, Morrell, Gareth, Trimnell, Cassandra
Published in Blood (02.11.2023)
Published in Blood (02.11.2023)
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Journal Article
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)
Jiménez-Yuste, Víctor, Oldenburg, Johannes, Tzeng, Eunice, Trzaskoma, Ben, Sanabria, Fabian, Mahlangu, Johnny
Published in Blood (23.11.2021)
Published in Blood (23.11.2021)
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Journal Article
A Randomized Trial of Factor VIII and Neutralizing Antibodies in Hemophilia A
Peyvandi, Flora, Mannucci, Pier M, Garagiola, Isabella, El-Beshlawy, Amal, Elalfy, Mohsen, Ramanan, Vijay, Eshghi, Peyman, Hanagavadi, Suresh, Varadarajan, Ramabadran, Karimi, Mehran, Manglani, Mamta V, Ross, Cecil, Young, Guy, Seth, Tulika, Apte, Shashikant, Nayak, Dinesh M, Santagostino, Elena, Mancuso, Maria Elisa, Sandoval Gonzalez, Adriana C, Mahlangu, Johnny N, Bonanad Boix, Santiago, Cerqueira, Monica, Ewing, Nadia P, Male, Christoph, Owaidah, Tarek, Soto Arellano, Veronica, Kobrinsky, Nathan L, Majumdar, Suvankar, Perez Garrido, Rosario, Sachdeva, Anupam, Simpson, Mindy, Thomas, Mathew, Zanon, Ezio, Antmen, Bulent, Kavakli, Kaan, Manco-Johnson, Marilyn J, Martinez, Monica, Marzouka, Esperanza, Mazzucconi, Maria G, Neme, Daniela, Palomo Bravo, Angeles, Paredes Aguilera, Rogelio, Prezotti, Alessandra, Schmitt, Klaus, Wicklund, Brian M, Zulfikar, Bulent, Rosendaal, Frits R
Published in The New England journal of medicine (26.05.2016)
Published in The New England journal of medicine (26.05.2016)
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Journal Article
Long‐term safety and efficacy of the anti‐tissue factor pathway inhibitor marstacimab in participants with severe haemophilia: Phase II study results
Mahlangu, Johnny, Luis Lamas, Jose, Cristobal Morales, Juan, Malan, Daniel R., Teeter, John, Charnigo, Robert J., Hwang, Eunhee, Arkin, Steven
Published in British journal of haematology (01.01.2023)
Published in British journal of haematology (01.01.2023)
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Journal Article
A Multiple Ascending Dose Study of Mim8 in Patients with Severe Hemophilia a with or without Factor VIII Inhibitors
Lentz, Steven R., Chowdary, Pratima, Lopez Jaime, Francisco Jose, Mahlangu, Johnny, Matytsina, Irina, Nielsen, Anne Louise, Windyga, Jerzy
Published in Blood (15.11.2022)
Published in Blood (15.11.2022)
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Journal Article
FRONTIER1: a partially randomized phase 2 study assessing the safety, pharmacokinetics, and pharmacodynamics of Mim8, a factor VIIIa mimetic
Lentz, Steven R., Chowdary, Pratima, Gil, Lidia, Lopez-Jaime, Francisco J., Mahlangu, Johnny, Matytsina, Irina, Nielsen, Anne Louise, Windyga, Jerzy
Published in Journal of thrombosis and haemostasis (01.04.2024)
Published in Journal of thrombosis and haemostasis (01.04.2024)
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Journal Article
The evolving landscape of gene therapy for congenital haemophilia: An unprecedented, problematic but promising opportunity for worldwide clinical studies
Spadarella, Gaia, Di Minno, Alessandro, Brunetti-Pierri, Nicola, Mahlangu, Johnny, Di Minno, Giovanni
Published in Blood reviews (01.03.2021)
Published in Blood reviews (01.03.2021)
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Journal Article
Marstacimab, an Anti-Tissue Factor Pathway Inhibitor, in Participants with Hemophilia Α or B, with and without Inhibitors: An Integrated Analysis of Safety
Acharya, Suchitra, Matino, Davide, Mahlangu, Johnny, Taylor, Carrie Turich, Hwang, Eunhee, Hinnershitz, Tina, Mefyod, Eugeny, Palladino, Andrew, Biondo, Francesca, Gould, Travis, Teeter, John
Published in Blood (02.11.2023)
Published in Blood (02.11.2023)
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Journal Article
Recombinant factor VIII Fc fusion protein for the treatment of severe haemophilia A: Final results from the ASPIRE extension study
Nolan, Beatrice, Mahlangu, Johnny, Pabinger, Ingrid, Young, Guy, Konkle, Barbara A., Barnes, Chris, Nogami, Keiji, Santagostino, Elena, Pasi, K. John, Khoo, Liane, Winding, Bent, Yuan, Huixing, Fruebis, Joachim, Rudin, Dan, Oldenburg, Johannes
Published in Haemophilia : the official journal of the World Federation of Hemophilia (01.05.2020)
Published in Haemophilia : the official journal of the World Federation of Hemophilia (01.05.2020)
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Journal Article
Two-Year Outcomes of Valoctocogene Roxaparvovec Therapy for Hemophilia A
Mahlangu, Johnny, Kaczmarek, Radoslaw, von Drygalski, Annette, Shapiro, Susan, Chou, Sheng-Chieh, Ozelo, Margareth C., Kenet, Gili, Peyvandi, Flora, Wang, Michael, Madan, Bella, Key, Nigel S., Laffan, Michael, Dunn, Amy L., Mason, Jane, Quon, Doris V., Symington, Emily, Leavitt, Andrew D., Oldenburg, Johannes, Chambost, Hervé, Reding, Mark T., Jayaram, Kala, Yu, Hua, Mahajan, Reena, Chavele, Konstantia-Maria, Reddy, Divya B., Henshaw, Joshua, Robinson, Tara M., Wong, Wing Yen, Pipe, Steven W.
Published in The New England journal of medicine (23.02.2023)
Published in The New England journal of medicine (23.02.2023)
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Journal Article
Core data set on safety, efficacy, and durability of hemophilia gene therapy for a global registry: Communication from the SSC of the ISTH
Konkle, Barbara, Pierce, Glen, Coffin, Donna, Naccache, Mayss, Clark, R. Cary, George, Lindsey, Iorio, Alfonso, O’Mahony, Brian, Pipe, Steven, Skinner, Mark, Watson, Crystal, Peyvandi, Flora, Mahlangu, Johnny
Published in Journal of thrombosis and haemostasis (01.11.2020)
Published in Journal of thrombosis and haemostasis (01.11.2020)
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Journal Article
Underdiagnosis of iron deficiency anaemia in HIV-infected individuals: a pilot study using soluble transferrin receptors and intensive bone marrow iron stores to improve the diagnosis
Mankgele, Mahlatse, Variava, Ebrahim, Moloantoa, Tumelo, Otwombe, Kennedy, Hlongwane, Khuthadzo, Disenyane, Dineo, Bida, Meshack, Chikwati, Raylton, Snyman, Tracy, Martinson, Neil, Mahlangu, Johnny
Published in Journal of clinical pathology (01.08.2023)
Published in Journal of clinical pathology (01.08.2023)
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Journal Article
Unexplained hemorrhagic syndrome? Consider acquired hemophilia A or B
Constantinescu, Catalin, Jitaru, Ciprian, Pasca, Sergiu, Dima, Delia, Dirzu, Noemi, Coriu, Daniel, Zdziarska, Joanna, Ghiaur, Gabriel, Mahlangu, Johnny, Tomuleasa, Ciprian
Published in Blood reviews (01.05.2022)
Published in Blood reviews (01.05.2022)
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Journal Article