Zobrazeno 1 - 10
of 64
pro vyhledávání: '"Adam Giermasz"'
Autor:
Annette Von Drygalski, Adam Giermasz, Giancarlo Castaman, Nigel S. Key, Susan Lattimore, Frank W.G. Leebeek, Wolfgang Miesbach, Michael Recht, Alison Long, Robert Gut, Eileen K. Sawyer, Steven W. Pipe
Publikováno v:
Blood Advances, Vol 3, Iss 21, Pp 3241-3247 (2019)
Etranacogene dezaparvovec (AMT-061) is a recombinant AAV5 vector including a gene cassette containing the factor IX (FIX) Padua variant under the control of a liver-specific promoter. A phase 2b study was conducted to confirm that a single dose of 2
Externí odkaz:
https://doaj.org/article/66cb66c72e9147b78eee95d74a7a1df4
Autor:
Amy L. Dunn, Yesim Dargaud, Yasmina L. Abajas, Manuel Carcao, Giancarlo Castaman, Adam Giermasz, Cédric Hermans, Magdalena D. Lewandowska, Johnny Mahlangu, Shannon L. Meeks, Wolfgang A. Miesbach, Michael Recht, Vanessa Salinas, Tammuella Chrisentery-Singleton, Hongying Wang, Ian S Mitchell, Guy Young
Publikováno v:
Blood. 140:8485-8488
Autor:
Margareth C, Ozelo, Johnny, Mahlangu, K John, Pasi, Adam, Giermasz, Andrew D, Leavitt, Michael, Laffan, Emily, Symington, Doris V, Quon, Jiaan-Der, Wang, Kathelijne, Peerlinck, Steven W, Pipe, Bella, Madan, Nigel S, Key, Glenn F, Pierce, Brian, O'Mahony, Radoslaw, Kaczmarek, Joshua, Henshaw, Adebayo, Lawal, Kala, Jayaram, Mei, Huang, Xinqun, Yang, Wing Y, Wong, Benjamin, Kim, Annette, Von Drygalski
Publikováno v:
New England Journal of Medicine. 386:1013-1025
BACKGROUND: Valoctocogene roxaparvovec (AAV5-hFVIII-SQ) is an adeno-associated virus 5 (AAV5)-based gene-therapy vector containing a coagulation factor VIII complementary DNA driven by a liver-selective promoter. The efficacy and safety of the therap
Autor:
Annette von Drygalski, Esteban Gomez, Adam Giermasz, Giancarlo Castaman, Nigel S Key, Susan S Lattimore, Frank W.G. Leebeek, Wolfgang A Miesbach, Michael Recht, Robert Z Gut, Ricardo Dolmetsch, Paul E Monahan, Sandra Le Quellec, Steven W Pipe
Publikováno v:
Blood advances. American Society of Hematology
Etranacogene dezaparvovec (AMT-061) is a recombinant adeno-associated virus serotype 5 (AAV5) vector containing a codon-optimized Padua variant human factor IX (FIX) transgene with a liver-specific promoter. Here we report 3-year outcomes from a Phas
Autor:
Jerzy Windyga, Ian S Mitchell, Yevhenii Averianov, Adam Giermasz, Maria Fernanda Lopez Fernandez, Laura Villarreal Martinez, W. Allan Alexander, Wolfgang Miesbach, Oleksandra Stasyshyn, Ismail Haroon Mitha, Daniel P. Hart, James V. Luck, Janna M. Journeycake, Miguel A. Escobar, Daniel Bonzo, Cindy A. Leissinger, Cédric Hermans, Mark T. Reding, J.-F. Schved, Kateryna V. Vilchevska, Craig M. Kessler, Jonathan M. Ducore, Doris Quon, Thomas A. Wilkinson, Johnny Mahlangu, Ahmad Al-Sabbagh, Michael Wang
Publikováno v:
Haemophilia, Vol. 27, no. 6, p. 911-920 (2021)
Introduction Surgical procedures in persons with haemophilia A or B with inhibitors (PwHABI) require the use of bypassing agents (BPA) and carry a high risk of complications. Historically, only two BPAs have been available; these are reported to have
Autor:
Adam Giermasz, Nathan Visweshwar, Thomas J. Harrington, Andrew D Leavitt, Barbara Konkle, Jeremy Rupon, Gregory Di Russo, Li-Jung Tseng, Maria de los Angeles Resa, Florence Ganne, Delphine Agathon, Frank Plonski, Didier Rouy, Bettina M. Cockroft, Annie Fang, Steven Arkin
Publikováno v:
Blood. 140:7776-7777
Autor:
Tammuella Chrisentery-Singleton, Lauren E. Amos, Daniel Bonzo, Miguel Escobar, Adam Giermasz, Emmanuelle Lagrue, Christine Knoll, Sonia Nasr, Michael Recht, Spencer Sullivan, Doris V. Quon, Mark T. Reding
Publikováno v:
Blood. 140:11360-11361
Autor:
Emily Symington, Glenn F. Pierce, Adam Giermasz, K. John Pasi, Stefan Tiefenbacher, Stephen J. Zoog, Mary Robinson, Savita Rangarajan, Steffen Rosen, Christian Vettermann, Donnie Mackenzie, Jaydeep K. Srimani, Benjamin Kim, Mei Huang, Terri Christianson
Publikováno v:
Blood. 136:2524-2534
Adeno-associated virus (AAV)-based gene therapies can restore endogenous factor VIII (FVIII) expression in hemophilia A (HA). AAV vectors typically use a B-domain–deleted FVIII transgene, such as human FVIII-SQ in valoctocogene roxaparvovec (AAV5-F
Autor:
Michael Recht, Wolfgang Miesbach, Nigel S. Key, Giancarlo Castaman, Esteban Gomez, Steven W. Pipe, Robert Gut, A. von Drygalski, Susan Lattimore, Adam Giermasz, Frank W.G. Leebeek
Publikováno v:
65th Annual Meeting of the Society of Thrombosis and Haemostasis Research.
Autor:
Tammuella Chrisentery-Singleton, Nikki Hirsh, Michael U. Callaghan, Miguel A. Escobar, W. Allan Alexander, Adam Giermasz, Daniel Bonzo, Sonia Nasr, Michael Recht, Doris Quon, Janna M. Journeycake, Ahmad Al-Sabbagh
Publikováno v:
Blood. 138:3201-3201
Background: A new recombinant activated factor VII, eptacog beta (SEVENFACT®, rFVIIa-jncw) has been approved by the U.S. Food and Drug Administration (FDA) for the treatment of bleeding events (BEs) in individuals >12 years of age with hemophilia A