Establishing the appropriate primary endpoint in haemophilia gene therapy pivotal studies
Author:
Affiliation:
1. National Hemophilia Foundation Medical and Scientific Advisory Council; New York NY USA
2. World Federation of Hemophilia; Montreal Quebec Canada
3. European Haemophilia Consortium; Brussels Belgium
Publisher
Wiley
Subject
Genetics (clinical),Hematology,General Medicine
Link
http://onlinelibrary.wiley.com/wol1/doi/10.1111/hae.13313/fullpdf
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2. The hemophilias-from royal genes to gene therapy. N Engl J Med. 2001; 344:1773-9. Review. No abstract available. Erratum;Mannucci;N Engl J Med,2001
3. Recent advances in the diagnosis and treatment of haemophilia;Biggs;Proc R Soc Med,1957
4. Clinical severity of haemophilia A: does the classification of the 1950s still stand?;Den;Haemophilia,2011
5. Factor IX and rare coagulation disorders of the Scientific and Standardization Committee of the International Society on Thrombosis and Hemostasis. Definitions in hemophilia: communication from the SSC of the ISTH;Blanchette;J Thromb Haemost,2014
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