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Haemophilia. 2017 Sep;23(5):643-644. doi: 10.1111/hae.13313. Epub 2017 Aug 17.

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

Author information

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.

KEYWORDS:

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

PMID:
28833947
DOI:
10.1111/hae.13313
[Indexed for MEDLINE]

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