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Establishing the appropriate primary endpoint in haemophilia gene therapy pivotal studies

G F Pierce1,2, M V Ragni1, H M van den Berg2

  • 1National Hemophilia Foundation Medical and Scientific Advisory Council, New York, NY, USA.

Haemophilia : the Official Journal of the World Federation of Hemophilia
|August 24, 2017
PubMed
Abstract

No abstract available in PubMed .

Keywords:
annualized bleeding rateclinical trialsfactor IXfactor VIIIgene therapyhaemophilia

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