Establishing the appropriate primary endpoint in haemophilia gene therapy pivotal studies.
作者信息
Pierce G F, Ragni M V, van den Berg H M, Weill A, O'Mahony B, Skinner M W, Pipe S W
机构信息
National Hemophilia Foundation Medical and Scientific Advisory Council, New York, NY, USA.
World Federation of Hemophilia, Montreal, Quebec, Canada.
出版信息
Haemophilia. 2017 Sep;23(5):643-644. doi: 10.1111/hae.13313. Epub 2017 Aug 17.
PMID:28833947
Abstract
摘要
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