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Latest News

New Gene Therapy Shows Promise in Hemophilia A Mouse Model, Study Shows

17 March 2020

 

 

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Human Gene Therapy for Hemophilia

04 February 2020

 

 

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High Dose of Gene Therapy for Hemophilia B Normalizing FIX Levels, Early Trial Data Show

18 February 2020

 

 

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BioMarin Submits Biologics License Application to U.S. Food and Drug Administration for Valoctocogene Roxaparvovec to Treat Hemophilia A

02 January 2020

 

 

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  • Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A
    02 January 2020
  • BioMarin submits EMA filing for first hemophilia gene therapy
    18 December 2019
  • Enhanced Factor IX Activity following Administration of AAV5-R338L "Padua" Factor IX versus AAV5 WT Human Factor IX in NHPs
    17 November 2019
  • Patient perspectives on novel treatments in hemophilia A: a qualitative study
    17 November 2019
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Supported by educational grants from BioMarin, Freeline Therapeutics Limited, Pfizer Inc., Shire, Spark Therapeutics, and uniQure, Inc

Click here to access the enduring symposium:
“Key Considerations: Advances in Gene Therapy for Hemophilia”

Featuring Glenn Pierce, MD, PhD, Lindsey George, MD, Alfonso Iorio, MD and Barbara Konkle, MD

© 2021 International Society on Thrombosis and Haemostasis, Inc. All rights reserved.
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    • 2020 Virtual Learning Sessions
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