Establishing the appropriate primary endpoint in haemophilia gene therapy pivotal studies-Reference-Cited by-同舟云学术

Establishing the appropriate primary endpoint in haemophilia gene therapy pivotal studies

Published:2017-08-17 Issue:5 Volume:23 Page:643-644
ISSN:1351-8216
Container-title:Haemophilia
language:en
Short-container-title:Haemophilia

Author:

Pierce G. F.¹²^ORCID,Ragni M. V.¹^ORCID,van den Berg H. M.²^ORCID,Weill A.²^ORCID,O'Mahony B.³^ORCID,Skinner M. W.¹,Pipe S. W.¹

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

Reference15 articles.

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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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2. Association of factor expression levels with health-related quality of life and direct medical costs for people with haemophilia B;Journal of Medical Economics;2022-03-15

3. Electronic diaries in the management of haemophilia gene therapy: Perspective of an expert group from the German, Austrian and Swiss Society on Thrombosis and Haemostasis (GTH);Haemophilia;2022-02-19

4. Delivery of AAV‐based gene therapy through haemophilia centres—A need for re‐evaluation of infrastructure and comprehensive care: A Joint publication of EAHAD and EHC;Haemophilia;2021-09-22

5. Gene therapy for hemophilia: a review on clinical benefit, limitations, and remaining issues;Blood;2021-07-07