Stable and durable factor IX levels in patients with hemophilia B over 3 years after etranacogene dezaparvovec gene therapy
von Drygalski, Annette, Gomez, Esteban, Giermasz, Adam, Castaman, Giancarlo, Key, Nigel S., Lattimore, Susan U., Leebeek, Frank W. G., Miesbach, Wolfgang A., Recht, Michael, Gut, Robert, Dolmetsch, Ricardo, Monahan, Paul E., Le Quellec, Sandra, Pipe, Steven W.
Published in Blood advances (10.10.2023)
Published in Blood advances (10.10.2023)
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Journal Article
Durability of Factor IX Activity and Bleeding Rate in People with Severe or Moderately Severe Hemophilia B after 5 Years of Follow-up in the Phase 1/2 Study of AMT-060, and after 3 Years of Follow-up in the Phase 2b and 2 Years of Follow-up in the Phase 3 Studies of Etranacogene Dezaparvovec (AMT-061)
Miesbach, Wolfgang A., Recht, Michael, Key, Nigel S., Sivamurthy, Krupa, Monahan, Paul E., Pipe, Steven W.
Published in Blood (15.11.2022)
Published in Blood (15.11.2022)
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Journal Article
Efficacy and Safety of BAY 94-9027 (Damoctocog Alfa Pegol) Prophylaxis in Patients with Severe Hemophilia a and Comorbidities: A Post Hoc Analysis of PROTECT VIII Data
Miesbach, Wolfgang A., Di Minno, Giovanni, Santagostino, Elena, Klamroth, Dr, Bayh, Inga, Soto, Amaryllis, Hermans, Cedric
Published in Blood (13.11.2019)
Published in Blood (13.11.2019)
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Journal Article
Durability of Bleeding Protection and Factor IX Activity Levels Are Demonstrated in Individuals with and without Adeno-Associated Virus Serotype 5 Neutralizing Antibodies (Titers <1:700) with Comparable Safety in the Phase 3 HOPE-B Clinical Trial of Etranacogene Dezaparvovec Gene Therapy for Hemophilia B
Pipe, Steven W., Leebeek, Frank W.G., Recht, Michael, Key, Nigel S., Lattimore, Susan, Castaman, Giancarlo, Cooper, David, Verweij, Stephanie, Dolmetsch, Ricardo, Tarrant, Jacqueline, Li, Yanyan, Monahan, Paul E., Miesbach, Wolfgang A.
Published in Blood (15.11.2022)
Published in Blood (15.11.2022)
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Journal Article
Adults with Severe or Moderately Severe Hemophilia B Receiving Etranacogene Dezaparvovec in the HOPE-B Phase 3 Clinical Trial Continue to Experience a Stable Increase in Mean Factor IX Activity Levels and Durable Hemostatic Protection after 24 Months’ Follow-up
Pipe, Steven W., Leebeek, Frank W.G., Recht, Michael, Key, Nigel S., Lattimore, Susan, Castaman, Giancarlo, Coppens, Michiel, Cooper, David, Gut, Robert, Slawka, Sergio, Verweij, Stephanie, Dolmetsch, Ricardo, Li, Yanyan, Monahan, Paul E., Miesbach, Wolfgang A.
Published in Blood (15.11.2022)
Published in Blood (15.11.2022)
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Journal Article
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
Dunn, Amy L., Dargaud, Yesim, Abajas, Yasmina L., Carcao, Manuel, Castaman, Giancarlo, Giermasz, Adam, Hermans, Cédric, Lewandowska, Magdalena D., Mahlangu, Johnny, Meeks, Shannon L., Miesbach, Wolfgang A., Recht, Michael, Salinas, Vanessa, Chrisentery-Singleton, Tammuella, Wang, Hongying, Mitchell, Ian S, Young, Guy
Published in Blood (15.11.2022)
Published in Blood (15.11.2022)
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Journal Article
First-in-Human Phase 1/2 Clinical Trial of SIG-001, an Innovative Shielded Cell Therapy Platform, for Hemophilia Α
Shapiro, Amy D, Konkle, Barbara A., Croteau, Stacy E., Miesbach, Wolfgang A., Hay, Charles Richard Morris, Kazmi, Rashid, Mihova, Marina, Rangarajan, Savita, Pasi, John
Published in Blood (05.11.2020)
Published in Blood (05.11.2020)
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Journal Article
AMT-060 Gene Therapy in Adults with Severe or Moderate-Severe Hemophilia B Confirm Stable FIX Expression and Durable Reductions in Bleeding and Factor IX Consumption for up to 5 Years
Leebeek, Frank W.G., Meijer, Karina, Coppens, Michiel, Kampmann, Peter, Klamroth, Robert, Schutgens, Roger, Castaman, Giancarlo, Seifried, Erhard, Schwaeble, Joachim, Bönig, Halvard, Sawyer, Eileen K, Miesbach, Wolfgang A.
Published in Blood (05.11.2020)
Published in Blood (05.11.2020)
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Journal Article
Etranacogene Dezaparvovec (AAV5-Padua hFIX variant), an Enhanced Vector for Gene Transfer in Adults with Severe or Moderate-Severe Hemophilia B: Two Year Data from a Phase 2b Trial
von Drygalski, Annette, Giermasz, Adam, Castaman, Giancarlo, Key, Nigel S., Lattimore, Susan U., Leebeek, Frank W.G., Miesbach, Wolfgang A., Recht, Michael, Gomez, Esteban, Gut, Robert, Pipe, Steven W.
Published in Blood (05.11.2020)
Published in Blood (05.11.2020)
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Journal Article
Acquired Factor X Deficiency in Systemic Light Chain Amyloidosis: Factor X Recovery after Bleeding Complications, Splenectomy, Chemotherapy and Remission
Toenges, Rosa, Steinmann, Juliane, Siegemund, Annelie, Serve, Hubert, Miesbach, Wolfgang A.
Published in Blood (08.12.2017)
Published in Blood (08.12.2017)
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Journal Article
First Data from the Phase 3 HOPE-B Gene Therapy Trial: Efficacy and Safety of Etranacogene Dezaparvovec (AAV5-Padua hFIX variant; AMT-061) in Adults with Severe or Moderate-Severe Hemophilia B Treated Irrespective of Pre-Existing Anti-Capsid Neutralizing Antibodies
Pipe, Steven W., Recht, Michael, Key, Nigel S., Leebeek, Frank W.G., Castaman, Giancarlo, Lattimore, Susan U., Van Der Valk, Paul, Peerlinck, Kathelijne, Coppens, Michiel, O'Connell, Niamh, Pasi, John, Kampmann, Peter, Meijer, Karina, von Drygalski, Annette, Young, Guy, Hermans, Cedric, Astermark, Jan, Klamroth, Robert, Lemons, Richard S., Visweshwar, Nathan, Crary, Shelley, Kazmi, Rashid, Symington, Emily, Escobar, Miguel A., Gomez, Esteban, Kruse-Jarres, Rebecca, Kotowski, Adam, Quon, Doris, Wang, Michael, Wheeler, Allison P., Sawyer, Eileen K, Verweij, Stephanie, Colletta, Valerie, Bajma, Naghmana, Gut, Robert, Miesbach, Wolfgang A.
Published in Blood (19.11.2020)
Published in Blood (19.11.2020)
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Journal Article
Factor VIII Activity and Risk of Bleeding in Acquired Hemophilia a: Results from the Gth-AH 01/2010 Study
Holstein, Katharina, Smith, Andrea, Knöbl, Paul, Dobbelstein, Christiane, Miesbach, Wolfgang A., Spannagl, Michael, Heinz, Jürgen, Scholz, Ute, Eichler, Hermann, Huth-Kühne, Angela, Klamroth, Robert, Koch, Armin, Tiede, Andreas
Published in Blood (08.12.2017)
Published in Blood (08.12.2017)
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Journal Article
Stable Elevations in FIX Activity and Reductions in Annualized Bleeding Rate over up to 2 Years of Follow-up of Adults with Severe or Moderate-Severe Hemophilia B Treated with AMT-060 (AAV5-hFIX) Gene Therapy
Leebeek, Frank W.G., Meijer, K, Coppens, Michiel, Kampmann, Peter, Klamroth, R, Schutgens, Roger, Castaman, Giancarlo, Seifried, Erhard, Schwaeble, J, Bonig, Halvard, Sawyer, Eileen K, Miesbach, Wolfgang A.
Published in Blood (08.12.2017)
Published in Blood (08.12.2017)
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Journal Article
Phase 1-2 Clinical Trial of a Recombinant AAV5 Vector Containing the Human FIX Gene in Patients with Severe or Moderately Severe Haemophilia B
Miesbach, Wolfgang A., Meyer, Christian, Nijmeijer, Bart, Salmon, Florence, Grosios, Nadina, Petry, Harald, Leebeek, Frank W.G.
Published in Blood (06.12.2014)
Published in Blood (06.12.2014)
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Journal Article
Stable and durable factor IX levels in hemophilia B patients over 3 years post etranacogene dezaparvovec gene therapy
von Drygalski, Annette, Gomez, Esteban, Giermasz, Adam, Castaman, Giancarlo, Key, Nigel S, Lattimore, Susan S, Leebeek, Frank W G, Miesbach, Wolfgang A, Recht, Michael, Gut, Robert Z, Dolmetsch, Ricardo, Monahan, Paul E, Le Quellec, Sandra, Pipe, Steven W
Published in Blood advances (09.12.2022)
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Published in Blood advances (09.12.2022)
Journal Article
Interim Results from a Dose Escalating Study of AMT-060 (AAV5-hFIX) Gene Transfer in Adult Patients with Severe Hemophilia B
Leebeek, Frank W.G., Tangelder, Marco, Meijer, Karina, Castaman, Giancarlo, Cattaneo, Federica, Coppens, Michiel, Kampmann, Peter, Klamroth, Robert, Schutgens, Roger E.G., Seifried, Erhard, Schwaeble, Joachim, Boenig, Halvard, Hendriks, Maaike, Corzo, Deya, Miesbach, Wolfgang A.
Published in Blood (02.12.2016)
Published in Blood (02.12.2016)
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Journal Article
Descriptive Evaluation of Age-Related Differences in Clinical Manifestation and Treatment of Von Willebrand’s Disease in an Open-Label, Prospective, Non-Interventional Study (wilate®-SET)
Hegener, Oliver, Scharrer, Inge, Feddern, Jennifer, V, Wolfgang A. Miesbach, Von Depka, Mario, Kadar, Janos
Published in Blood (16.11.2012)
Published in Blood (16.11.2012)
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Journal Article