Disease and treatment burden of patients with haemophilia entering the explorer6 non‐interventional study
Windyga, Jerzy, Apte, Shashikant, Frei‐Jones, Melissa, Fujii, Teruhisa, Lyu, Chuhl Joo, Villarreal Martinez, Laura, Sathar, Jameela, Stasyshyn, Oleksandra, Tran, Huyen, Zozulya, Nadezhda, Brown Frandsen, Renée, Neergaard, Jesper Skov, Thaung Zaw, Jay Jay, Mahlangu, Johnny
Published in European journal of haematology (01.11.2024)
Published in European journal of haematology (01.11.2024)
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Journal Article
The translational gap for gene therapies in low- and middle-income countries
Doxzen, Kevin W, Adair, Jennifer E, Fonseca Bazzo, Yris Maria, Bukini, Daima, Cornetta, Kenneth, Dalal, Varsha, Guerino-Cunha, Renato Luiz, Hongeng, Suradej, Jotwani, Geeta, Kityo-Mutuluuza, Cissy, Lakshmanan, Krishnamurti, Mahlangu, Johnny, Makani, Julie, Mathews, Vikram, Ozelo, Margareth C, Rangarajan, Savita, Scholefield, Janine, Batista Silva Júnior, João, McCune, Joseph M
Published in Science translational medicine (08.05.2024)
Published in Science translational medicine (08.05.2024)
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Journal Article
Three-year outcomes of valoctocogene roxaparvovec gene therapy for hemophilia A
Madan, Bella, Ozelo, Margareth C., Raheja, Priyanka, Symington, Emily, Quon, Doris V., Leavitt, Andrew D., Pipe, Steven W., Lowe, Gillian, Kenet, Gili, Reding, Mark T., Mason, Jane, Wang, Michael, von Drygalski, Annette, Klamroth, Robert, Shapiro, Susan, Chambost, Hervé, Dunn, Amy L., Oldenburg, Johannes, Chou, Sheng-Chieh, Peyvandi, Flora, Millar, Carolyn M., Osmond, Dane, Yu, Hua, Dashiell-Aje, Ebony, Robinson, Tara M., Mahlangu, Johnny
Published in Journal of thrombosis and haemostasis (01.07.2024)
Published in Journal of thrombosis and haemostasis (01.07.2024)
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Journal Article
Interim Analysis of the Extension Study with rVIII-SingleChain in Previously Untreated Patients (PUPs) with Severe Hemophilia A (CSL627-3001)
Santagostino, Elena, Fischer, Kathelijn, Koenigs, Christoph, Djambas Khayat, Claudia, Lucas, Samantha, Salazar, Blanca, Brainsky, Andres, Chung, Thomas, Goldstein, Brahm, Mahlangu, Johnny
Published in Blood (13.11.2019)
Published in Blood (13.11.2019)
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Journal Article
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
Callaghan, Michael, Trzaskoma, Benjamin, Ko, Richard H., Lee, Lucy, Patel, Anisha M., Tzeng, Eunice, Shah, Michelle Lynn, Chang, Tiffany Y., Niggli, Markus, Dhalluin, Christophe, Mahlangu, Johnny
Published in Blood (13.11.2019)
Published in Blood (13.11.2019)
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Journal Article
The effect of emicizumab prophylaxis on long‐term, self‐reported physical health in persons with haemophilia A without factor VIII inhibitors in the HAVEN 3 and HAVEN 4 studies
Skinner, Mark W., Négrier, Claude, Paz‐Priel, Ido, Chebon, Sammy, Jiménez‐Yuste, Victor, Callaghan, Michael U., Lehle, Michaela, Niggli, Markus, Mahlangu, Johnny, Shapiro, Amy, Shima, Midori, Campinha‐Bacote, Avrita, Levy, Gallia G., Oldenburg, Johannes, Mackensen, Sylvia, Pipe, Steven W.
Published in Haemophilia : the official journal of the World Federation of Hemophilia (01.09.2021)
Published in Haemophilia : the official journal of the World Federation of Hemophilia (01.09.2021)
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Journal Article
Health-related quality of life following valoctocogene roxaparvovec gene therapy for severe hemophilia A in the phase 3 trial GENEr8-1
O'Mahony, Brian, Dunn, Amy L, Leavitt, Andrew D, Peyvandi, Flora, Ozelo, Margareth C, Mahlangu, Johnny, Peerlinck, Kathelijne, Wang, Jiaan-Der, Lowe, Gillian C, Tan, Chee Wee, Giermasz, Adam, Tran, Huyen, Khoo, Teh-Liane, Cockrell, Erin, Pepperell, Dominic, Chambost, Hervé, López Fernández, Maria Fernanda, Kazmi, Rashid, Majerus, Elaine, Skinner, Mark W, Klamroth, Robert, Quinn, Jennifer, Yu, Hua, Wong, Wing Yen, Robinson, Tara M, Pipe, Steven W
Published in Journal of thrombosis and haemostasis (01.12.2023)
Published in Journal of thrombosis and haemostasis (01.12.2023)
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Journal Article
RNA sequencing suggests that non‐coding RNAs play a role in the development of acquired haemophilia
Tigu, Adrian Bogdan, Hotea, Ionut, Drula, Rares, Zimta, Alina‐Andreea, Dirzu, Noemi, Santa, Maria, Constantinescu, Catalin, Dima, Delia, Bergthorsson, Jon Thor, Greiff, Victor, Gulei, Diana, Coriu, Daniel, Serban, Margit, Mahlangu, Johnny, Tomuleasa, Ciprian
Published in Journal of cellular and molecular medicine (01.07.2023)
Published in Journal of cellular and molecular medicine (01.07.2023)
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Journal Article
Health‐related quality of life and health status in persons with haemophilia A with inhibitors: A prospective, multicentre, non‐interventional study (NIS)
Mahlangu, Johnny, Oldenburg, Johannes, Callaghan, Michael U., Shima, Midori, Mancuso, Maria Elisa, Trask, Peter, Recht, Michael, Garcia, Claudia, Yang, Renchi, Lehle, Michaela, Macharia, Harrison, Asikanius, Elina, Levy, Gallia G., Kruse‐Jarres, Rebecca, von Mackensen, Sylvia
Published in Haemophilia : the official journal of the World Federation of Hemophilia (01.05.2019)
Published in Haemophilia : the official journal of the World Federation of Hemophilia (01.05.2019)
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Journal Article
Emicizumab is well tolerated and effective in people with congenital hemophilia A regardless of age, severity of disease, or inhibitor status: a scoping review
Young, Guy, Pipe, Steven W., Kenet, Gili, Oldenburg, Johannes, Safavi, Mariam, Czirok, Tuende, Nissen, Francis, Mahlangu, Johnny
Published in Research and practice in thrombosis and haemostasis (01.05.2024)
Published in Research and practice in thrombosis and haemostasis (01.05.2024)
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Journal Article
Emicizumab in people with moderate or mild haemophilia A (HAVEN 6): a multicentre, open-label, single-arm, phase 3 study
Négrier, Claude, Mahlangu, Johnny, Lehle, Michaela, Chowdary, Pratima, Catalani, Olivier, Bernardi, Ronald J, Jiménez-Yuste, Víctor, Beckermann, Benjamin M, Schmitt, Christophe, Ventriglia, Giuliana, Windyga, Jerzy, d'Oiron, Roseline, Moorehead, Paul, Koparkar, Sunita, Teodoro, Vanda, Shapiro, Amy D, Oldenburg, Johannes, Hermans, Cedric
Published in The Lancet. Haematology (01.03.2023)
Published in The Lancet. Haematology (01.03.2023)
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Journal Article
Health-Related Quality of Life Following Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A in the Phase 3 Trial GENEr8-1
O'Mahony, Brian, Mahlangu, Johnny, Peerlinck, Kathelijne, Wang, Jiaan-Der, Lowe, Gillian, Tan, Chee Wee, Giermasz, Adam, Tran, Huyen A, Khoo, Teh-Liane, Cockrell, Erin, Pepperell, Dominic, Chambost, Hervé, López Fernández, María Fernanda, Kazmi, Rashid, Majerus, Elaine, Skinner, Mark W, Klamroth, Robert, Quinn, Jennifer, Yu, Hua, Wong, Wing Yen, Lawal, Adebayo, Robinson, Tara M, Kim, Benjamin
Published in Blood (23.11.2021)
Published in Blood (23.11.2021)
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Journal Article
Bleeding and safety outcomes in persons with haemophilia A without inhibitors: Results from a prospective non‐interventional study in a real‐world setting
Kruse‐Jarres, Rebecca, Oldenburg, Johannes, Santagostino, Elena, Shima, Midori, Kempton, Christine L., Kessler, Craig M., Lehle, Michaela, Chebon, Sammy, Selak Bienz, Nives, Asikanius, Elina, Mahlangu, Johnny
Published in Haemophilia : the official journal of the World Federation of Hemophilia (01.03.2019)
Published in Haemophilia : the official journal of the World Federation of Hemophilia (01.03.2019)
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Journal Article
B-YOND Final Results Confirm Established Safety, Sustained Efficacy, and Extended Dosing Interval for Up to 4 Years of Treatment With rFIXFc in Previously Treated Subjects With Severe Hemophilia B
Ragni, Margaret, Kulkarni, Roshni, Pasi, K. John, Fischer, Kathelijn, Mahlangu, Johnny, Shapiro, Amy, Nolan, Beatrice, Oldenburg, Johannes, Matsushita, Tadashi, Willemze, Annemieke, Yuan, Huixing, Rudin, Dan
Published in Blood (29.11.2018)
Published in Blood (29.11.2018)
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Journal Article
ASPIRE Final Results Confirm Established Safety and Sustained Efficacy for Up to 4 Years of Treatment With rFVIIIFc in Previously Treated Subjects With Severe Hemophilia A
Nolan, Beatrice, Mahlangu, Johnny, Young, Guy, Konkle, Barbara A, Pasi, K. John, Oldenburg, Johannes, Nogami, Keiji, Tripkovic, Nikola, Yuan, Huixing, Rudin, Dan
Published in Blood (29.11.2018)
Published in Blood (29.11.2018)
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Journal Article
Prophylaxis vs. on‐demand treatment with BAY 81‐8973, a full‐length plasma protein‐free recombinant factor VIII product: results from a randomized trial (LEOPOLD II)
Kavakli, K., Yang, R., Rusen, L., Beckmann, H., Tseneklidou‐Stoeter, D., Maas Enriquez, M., Yang, Renchi, Zhao, Yongqiang, Sun, Jing, Wang, Xuefeng, Wu, Depei, Hlusi, Antonin, Fukutake, Katsuyuki, Hanabusa, Hideji, Fujii, Teruhisa, Ramírez, Oscar Pérez, Alvarado, Blanca Salazar, Serban, Margit, Rusen, Luminita, Uscatescu, Valentina, Truica, Cristina, Kostic, Gordana, Konstantinidis, Nada, Igrutinovic, Zoran, Perina, Farida, Andreeva, Tatiana, Kavakli, Kaan, Antmen, Bulent, Sasmaz, Ilgen, Kupesiz, Alphan, Yesilipek, Mehmet Akif, Peng, Ching‐Tien, French, James, Escobar, Miguel, Mahlangu, Johnny, Pool, Roger
Published in Journal of thrombosis and haemostasis (01.03.2015)
Published in Journal of thrombosis and haemostasis (01.03.2015)
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Journal Article
BAY 81‐8973 demonstrated efficacy, safety and joint status improvement in patients with severe haemophilia A in the LEOPOLD I extension for ≤2 years
Mahlangu, Johnny, Lopez Fernandez, Maria Fernanda, Santagostino, Elena, Lalezari, Shadan, Tseneklidou‐Stoeter, Despina, Beckmann, Horst, Church, Nikki
Published in European journal of haematology (01.06.2020)
Published in European journal of haematology (01.06.2020)
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Journal Article
Nonacog beta pegol (N9-GP) in haemophilia B: A multinational phase III safety and efficacy extension trial (paradigm™4)
Young, Guy, Collins, Peter W, Colberg, Torben, Chuansumrit, Ampaiwan, Hanabusa, Hideji, Lentz, Steven R, Mahlangu, Johnny, Mauser-Bunschoten, Evelien P, Négrier, Claude, Oldenburg, Johannes, Patiroglu, Turkan, Santagostino, Elena, Tehranchi, Ramin, Zak, Marek, Karim, Faraizah Abdul
Published in Thrombosis research (01.05.2016)
Published in Thrombosis research (01.05.2016)
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Journal Article
Long-term safety and efficacy of extended-interval prophylaxis with recombinant factor IX Fc fusion protein (rFIXFc) in subjects with haemophilia B
Pasi, K John, Fischer, Kathelijn, Ragni, Margaret, Nolan, Beatrice, Perry, David J, Kulkarni, Roshni, Ozelo, Margareth, Mahlangu, Johnny, Shapiro, Amy D, Baker, Ross I, Bennett, Carolyn M, Barnes, Christopher, Oldenburg, Johannes, Matsushita, Tadashi, Yuan, Huixing, Ramirez-Santiago, Alejandra, Pierce, Glenn F, Allen, Geoffrey, Mei, Baisong
Published in Thrombosis and haemostasis (2017)
Published in Thrombosis and haemostasis (2017)
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Journal Article
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
Young, Guy, Pipe, Steven W, Hermans, Cédric, Carcao, Manuel, Castaman, Giancarlo, Davis, Joanna A., Ducore, Jonathan M., Dunn, Amy L., Escobar, Miguel A., Journeycake, Janna, Khan, Osman, Mahlangu, Johnny, Meeks, Shannon, Mitha, Ismail Haroon, Négrier, Claude, Nowak-Göttl, Ulrike, Recht, Michael, Chrisentery-Singleton, Tammuella, Stasyshyn, Oleksandra, Vilchevska, Kateryna V., Villarreal Martinez, Laura, Wang, Michael, Windyga, Jerzy, Alexander, W. Allan, Al-Sabbagh, Ahmad, Bonzo, Daniel, Macie, Christopher, Mitchell, Ian S., Wilkinson, Thomas A., Shapiro, Amy D.
Published in Blood (23.11.2021)
Published in Blood (23.11.2021)
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