Measuring success in hemophilia gene therapy using a factor level & outcomes yardstick
Author:
Affiliation:
1. Department of Medicine, University of Pittsburgh Medical Center, Pittsburgh, PA, USA
2. Hemophilia Center of Western Pennsylvania, Pittsburgh, PA, USA
Publisher
Informa UK Limited
Subject
Hematology
Link
https://www.tandfonline.com/doi/pdf/10.1080/17474086.2018.1423961
Reference15 articles.
1. Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B
2. Clinical severity of haemophilia A: does the classification of the 1950s still stand?
3. Establishing the appropriate primary endpoint in haemophilia gene therapy pivotal studies
4. Assessments of outcome in haemophilia – a patient perspective
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