Establishing the appropriate primary endpoint in haemophilia gene therapy pivotal studies

Haemophilia. 2017 Sep;23(5):643-644. doi: 10.1111/hae.13313. Epub 2017 Aug 17.
No abstract available

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

Publication types

  • Editorial

MeSH terms

  • Endpoint Determination / methods*
  • Genetic Therapy*
  • Hemophilia A / genetics*
  • Hemophilia A / therapy*
  • Humans