Journal article
Core outcome set for gene therapy in haemophilia: Results of the coreHEM multistakeholder project
Abstract
BACKGROUND: Gene therapy trial results show potential to cure haemophilia A and haemophilia B. Securing broad access to a cure for a lifelong chronic disease is anticipated to face barriers at the individual and healthcare system levels, which can be partly mitigated by harmonized planning of clinical research studies. The aim of the coreHEM project was to determine the set of outcome measures required to evaluate efficacy, safety, comparative …
Authors
Iorio A; Skinner MW; Clearfield E; Messner D; Pierce GF; Witkop M; Tunis S; panel TC
Journal
Haemophilia, Vol. 24, No. 4, pp. e167–e172
Publisher
Wiley
Publication Date
7 2018
DOI
10.1111/hae.13504
ISSN
1351-8216