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Establishing the appropriate primary endpoint in haemophilia gene therapy pivotal studies
Author(s) -
Pierce G. F.,
Ragni M. V.,
Berg H. M.,
Weill A.,
O'Mahony B.,
Skinner M. W.,
Pipe S. W.
Publication year - 2017
Publication title -
haemophilia
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.213
H-Index - 92
eISSN - 1365-2516
pISSN - 1351-8216
DOI - 10.1111/hae.13313
Subject(s) - haemophilia , medicine , clinical endpoint , clotting factor , genetic enhancement , dosing , clinical trial , factor ix , disease , haemophilia b , haemophilia a , intensive care medicine , bioinformatics , oncology , pediatrics , gene , genetics , biology
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