未登录 Prophylaxis with a recombinant factor VIII Fc in hemophilia A: long-term follow-up on joint health, efficacy, and safety from phase 3 studies in children and adults 重组因子VIII Fc预防血友病a: 儿童和成人3期研究关节健康、疗效和安全性的长期随访 Konkle, Barbara A.; Oldenburg, Johannes; Pasi, John; Kulkarni, Roshni; Nolan, Beatrice; Mahlangu, Johnny; Young, Guy; Brown, Simon A.; Pabinger, Ingrid; Shapiro, Amy; Negrier, Claude; Blanchette, Victor; Ragni, Margaret, V; Dumont, Jennifer; Lethagen, Stefan 分享 收藏
Post hoc longitudinal assessment of the efficacy and safety of recombinant factor IX Fc fusion protein in hemophilia B Shapiro, Amy D.; Kulkarni, Roshni; Ragni, Margaret V.; Chambost, Herve; Mahlangu, Johnny; Oldenburg, Johannes; Nolan, Beatrice; Ozelo, Margareth C.; Foster, Meredith C.; Willemze, Annemieke; Barnowski, Christopher; Jain, Nisha; Winding, Bent; Dumont, Jennifer; Lethagen, Stefan; Barnes, Chris; Pasi, K. John 分享 收藏
Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B Pipe, S. W.; Leebeek, F. W. G.; Recht, M.; Key, N. S.; Castaman, G.; Miesbach, W.; Lattimore, S.; Peerlinck, K.; Van der Valk, P.; Coppens, M.; Kampmann, P.; Meijer, K.; O'Connell, N.; Pasi, K. J.; Hart, D. P.; Kazmi, R.; Astermark, J.; Hermans, C. R. J. R.; Klamroth, R.; Lemons, R.; Visweshwar, N.; von Drygalski, A.; Young, G.; Crary, S. E.; Escobar, M.; Gomez, E.; Kruse-Jarres, R.; Quon, D. V.; Symington, E.; Wang, M.; Wheeler, A. P.; Gut, R.; Liu, Y. P.; Dolmetsch, R. E.; Cooper, D. L.; Li, Y.; Goldstein, B.; Monahan, P. E. 分享 收藏
Humanistic burden of problem joints for children and adults with haemophilia Burke, Tom; Rodriguez-Santana, Idaira; Chowdary, Pratima; Curtis, Randall; Khair, Kate; Laffan, Michael; Mclaughlin, Paul; Noone, Declan; O'Mahony, Brian; Pasi, John; Skinner, Mark; O'Hara, Jamie 分享 收藏
Managing surgery in hemophilia with recombinant factor VIII Fc and factor IX Fc: Data on safety and effectiveness from phase 3 pivotal studies 使用重组因子VIII Fc和因子IX Fc管理血友病的手术: 来自3期关键研究的安全性和有效性数据 Chowdary, Pratima; Holmstrom, Margareta; Mahlangu, Johnny N.; Ozelo, Margaret C.; Pabinger, Ingrid; Pasi, K. John; Ragni, Margaret, V; Shapiro, Amy; Barnowski, Chris; Lethagen, Stefan 分享 收藏
Interindividual variability in transgene mRNA and protein production following adeno-associated virus gene therapy for hemophilia A 血友病A的腺相关病毒基因治疗后转基因mRNA和蛋白质产生的个体间差异 Fong, Sylvia; Yates, Bridget; Sihn, Choong-Ryoul; Mattis, Aras N.; Mitchell, Nina; Liu, Su; Russell, Chris B.; Kim, Benjamin; Lawal, Adebayo; Rangarajan, Savita; Lester, Will; Bunting, Stuart; Pierce, Glenn F.; Pasi, K. John; Wong, Wing Yen 分享 收藏
Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A Ozelo, M. C.; Mahlangu, J.; Pasi, K. J.; Giermasz, A.; Leavitt, A. D.; Laffan, M.; Symington, E.; Quon, D., V; Wang, J-D; Peerlinck, K.; Pipe, S. W.; Madan, B.; Key, N. S.; Pierce, G. F.; O'Mahony, B.; Kaczmarek, R.; Henshaw, J.; Lawal, A.; Jayaram, K.; Huang, M.; Yang, X.; Wong, W. Y.; Kim, B. 分享 收藏
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Persistence of haemostatic response following gene therapy with valoctocogene roxaparvovec in severe haemophilia A Pasi, K. John; Laffan, Michael; Rangarajan, Savita; Robinson, Tara M.; Mitchell, Nina; Lester, Will; Symington, Emily; Madan, Bella; Yang, Xinqun; Kim, Benjamin; Pierce, Glenn F.; Wong, Wing Yen 分享 收藏
Targeting of antithrombin in hemophilia A or B with investigational siRNA therapeutic fitusiran-Results of the phase 1 inhibitor cohort Pasi, K. John; Lissitchkov, Toshko; Mamonov, Vasily; Mant, Tim; Timofeeva, Margarita; Bagot, Catherine; Chowdary, Pratima; Georgiev, Pencho; Gercheva-Kyuchukova, Liana; Madigan, Kate; Huy Van Nguyen; Yu, Qifeng; Mei, Baisong; Benson, Craig C.; Ragni, Margaret, V 分享 收藏
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Lupus Anticoagulant and Abnormal Coagulation Tests in Patients with Covid-19 Bowles, Louise; Platton, Sean; Yartey, Nada; Dave, Minal; Lee, Kurtis; Hart, Daniel P.; MacDonald, Vickie; Green, Laura; Sivapalaratnam, Suthesh; Pasi, K. John; MacCallum, Peter 分享 收藏
Long-term safety and sustained efficacy for up to 5 years of treatment with recombinant factor IX Fc fusion protein in subjects with haemophilia B: Results from the B-YOND extension study 在患有血友病B的受试者中使用重组因子IX Fc融合蛋白治疗长达5年的长期安全性和持续疗效: 来自b-yond扩展研究的结果 Pasi, K. John; Fischer, Kathelijn; Ragni, Margaret; Kulkarni, Roshni; Ozelo, Margareth C.; Mahlangu, Johnny; Shapiro, Amy; P'Ng, Stephanie; Chambost, Herve; Nolan, Beatrice; Bennett, Carolyn; Matsushita, Tadashi; Winding, Bent; Fruebis, Joachim; Yuan, Huixing; Rudin, Dan; Oldenburg, Johannes 分享 收藏
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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 分享 收藏
Towards a global multidisciplinary consensus framework on haemophilia gene therapy: Report of the 2nd World Federation of Haemophilia Gene Therapy Round Table 关于血友病基因治疗的全球多学科共识框架: 第二届世界血友病基因治疗联合会圆桌会议的报告 Pierce, Glenn F.; Pasi, K. John; Coffin, Donna; Kaczmarek, Radoslaw; Lillicrap, David; Mahlangu, Johnny; Rottellini, Dawn; Sannie, Thomas; Srivastava, Alok; VandenDriessche, Thierry; Weill, Alain 分享 收藏
Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A Pasi, K. John; Rangarajan, Savita; Mitchell, Nina; Lester, Will; Symington, Emily; Madan, Bella; Laffan, Michael; Russell, Chris B.; Li, Mingjin; Pierce, Glenn F.; Wong, Wing Y. 分享 收藏
Efficacy and safety of simoctocog alfa (Nuwiq®) in patients with severe hemophilia A: a review of clinical trial data from the GENA program Lissitchkov, Toshko; Klukowska, Anna; Pasi, John; Kessler, Craig M.; Klamroth, Robert; Liesner, Raina J.; Belyanskaya, Larisa; Walter, Olaf; Knaub, Sigurd; Bichler, Johann; Jansen, Martina; Oldenburg, Johannes 分享 收藏
Adenovirus-associated antibodies in UK cohort of hemophilia patients: A seroprevalence study of the presence of adenovirus-associated virus vector-serotypes AAV5 and AAV8 neutralizing activity and antibodies in patients with hemophilia A Stanford, Sophia; Pink, Ruth; Creagh, Desmond; Clark, Amanda; Lowe, Gillian; Curry, Nicola; Pasi, John; Perry, David; Fong, Sylvia; Hayes, Gregory; Chandrakumaran, Kandiah; Rangarajan, Savita 分享 收藏
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