Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A von 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

Volltext

AAV5–Factor VIII Gene Transfer in Severe Hemophilia A von Rangarajan, Savita, Walsh, Liron, Lester, Will, Perry, David, Madan, Bella, Laffan, Michael, Yu, Hua, Vettermann, Christian, Pierce, Glenn F, Wong, Wing Y, Pasi, K. John

Volltext

Interindividual variability in transgene mRNA and protein production following adeno-associated virus gene therapy for hemophilia A von 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

Volltext

Targeting of Antithrombin in Hemophilia A or B with RNAi Therapy von Pasi, K. John, Rangarajan, Savita, Georgiev, Pencho, Mant, Tim, Creagh, Michael D, Lissitchkov, Toshko, Bevan, David, Austin, Steve, Hay, Charles R, Hegemann, Inga, Kazmi, Rashid, Chowdary, Pratima, Gercheva-Kyuchukova, Liana, Mamonov, Vasily, Timofeeva, Margarita, Soh, Chang-Heok, Garg, Pushkal, Vaishnaw, Akshay, Akinc, Akin, Sørensen, Benny, Ragni, Margaret V

Volltext

Activity of transgene-produced B-domain–deleted factor VIII in human plasma following AAV5 gene therapy von Rosen, Steffen, Tiefenbacher, Stefan, Robinson, Mary, Huang, Mei, Srimani, Jaydeep, Mackenzie, Donnie, Christianson, Terri, Pasi, K. John, Rangarajan, Savita, Symington, Emily, Giermasz, Adam, Pierce, Glenn F., Kim, Benjamin, Zoog, Stephen J., Vettermann, Christian

Volltext

Functional definition and characterization of acute traumatic coagulopathy von Davenport, Ross, Manson, Joanna, DeʼAth, Henry, Platton, Sean, Coates, Amy, Allard, Shubha, Hart, Daniel, Pearse, Rupert, Pasi, K. John, MacCallum, Peter, Stanworth, Simon, Brohi, Karim

Volltext

Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A von Mahlangu, Johnny, Powell, Jerry S., Ragni, Margaret V., Chowdary, Pratima, Josephson, Neil C., Pabinger, Ingrid, Hanabusa, Hideji, Gupta, Naresh, Kulkarni, Roshni, Fogarty, Patrick, Perry, David, Shapiro, Amy, Pasi, K. John, Apte, Shashikant, Nestorov, Ivan, Jiang, Haiyan, Li, Shuanglian, Neelakantan, Srividya, Cristiano, Lynda M., Goyal, Jaya, Sommer, Jurg M., Dumont, Jennifer A., Dodd, Nigel, Nugent, Karen, Vigliani, Gloria, Luk, Alvin, Brennan, Aoife, Pierce, Glenn F.

Volltext

Diagnostic accuracy study of a factor VIII ELISA for detection of factor VIII antibodies in congenital and acquired haemophilia A von Batty, Paul, Moore, Gary W., Platton, Sean, Maloney, James C., Palmer, Ben, Bowles, Louise, Pasi, John K., Rangarajan, Savita, Hart, Daniel P.

Volltext

Lupus Anticoagulant and Abnormal Coagulation Tests in Patients with Covid-19 von Bowles, Louise, Platton, Sean, Yartey, Nada, Dave, Minal, Lee, Kurtis, Hart, Daniel P, MacDonald, Vickie, Green, Laura, Sivapalaratnam, Suthesh, Pasi, K. John, MacCallum, Peter

Volltext

Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A von 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

Volltext

Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A von 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

Volltext

Lupus Anticoagulant in Patients with Covid-19. Reply von Platton, Sean, Bowles, Louise, Pasi, K John

Volltext

Managing surgery in hemophilia with recombinant factor VIII Fc and factor IX Fc: Data on safety and effectiveness from phase 3 pivotal studies von Chowdary, Pratima, Holmström, Margareta, Mahlangu, Johnny N., Ozelo, Margaret C., Pabinger, Ingrid, Pasi, K. John, Ragni, Margaret V., Shapiro, Amy, Barnowski, Chris, Lethagen, Stefan

Volltext

Persistence of haemostatic response following gene therapy with valoctocogene roxaparvovec in severe haemophilia A von 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

Volltext

Targeting of antithrombin in hemophilia A or B with investigational siRNA therapeutic fitusiran—Results of the phase 1 inhibitor cohort von Pasi, K. John, Lissitchkov, Toshko, Mamonov, Vasily, Mant, Tim, Timofeeva, Margarita, Bagot, Catherine, Chowdary, Pratima, Georgiev, Pencho, Gercheva‐Kyuchukova, Liana, Madigan, Kate, Nguyen, Huy, Yu, Qifeng, Mei, Baisong, Benson, Craig C., Ragni, Margaret V.

Volltext

Recombinant factor VIII Fc for the treatment of haemophilia A von Hermans, Cedric, Mancuso, Maria Elisa, Nolan, Beatrice, Pasi, K. John

Volltext

Recombinant factor VIII Fc fusion protein for the treatment of severe haemophilia A: Final results from the ASPIRE extension study von 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

Volltext

Evolution of haemophilia integrated care in the era of gene therapy: Treatment centre’s readiness in United States and EU von Miesbach, Wolfgang, Pasi, K. John, Pipe, Steven W., Hermans, Cedric, O'Mahony, Brian, Guelcher, Christine, Steiner, Bruno, Skinner, Mark W.

Volltext

The safety and efficacy of recombinant human blood coagulation factor IX in previously untreated patients with severe or moderately severe hemophilia B von Shapiro, Amy D., Paola, Jorge Di, Cohen, Alice, Pasi, K. John, Heisel, Margaret A., Blanchette, Victor S., Abshire, Thomas C., Hoots, W. Keith, Lusher, Jeanne M., Negrier, Claude, Rothschild, Chantal, Roth, David A.

Volltext

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 extensio... von Pasi, K. John, Fischer, Kathelijn, Ragni, Margaret, Kulkarni, Roshni, Ozelo, Margareth C., Mahlangu, Johnny, Shapiro, Amy, P’Ng, Stephanie, Chambost, Hervé, Nolan, Beatrice, Bennett, Carolyn, Matsushita, Tadashi, Winding, Bent, Fruebis, Joachim, Yuan, Huixing, Rudin, Dan, Oldenburg, Johannes

Volltext