Literature DB >> 28833947

Establishing the appropriate primary endpoint in haemophilia gene therapy pivotal studies.

G F Pierce1,2, M V Ragni1, H M van den Berg2, A Weill2, B O'Mahony3, M W Skinner1, S W Pipe1.   

Abstract

Entities:  

Keywords:  annualized bleeding rate; clinical trials; factor IX; factor VIII; gene therapy; haemophilia

Mesh：

Year:  2017        PMID： 28833947     DOI： 10.1111/hae.13313

Source DB:  PubMed          Journal:  Haemophilia        ISSN： 1351-8216            Impact factor:   4.287


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  2 in total

1.  Annual Bleeding Rates: Pitfalls of Clinical Trial Outcomes in Hemophilia Patients.

Authors:  Christine Keipert; Mirco Müller-Olling; Franca Gauly; Cornelia Arras-Reiter; Anneliese Hilger
Journal:  Clin Transl Sci       Date:  2020-05-30       Impact factor: 4.689

2.  Clinical outcomes in hemophilia: Towards development of a core set of standardized outcome measures for research.

Authors:  Saunya Dover; Victor S Blanchette; Alok Srivastava; Kathelijn Fischer; Audrey Abad; Brian M Feldman
Journal:  Res Pract Thromb Haemost       Date:  2020-04-09
  2 in total

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