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Hemophilia resources

Core outcome set for gene therapy in haemophilia: Results of the coreHEM multistakeholder project
Iorio A, Skinner MW, Clearfield E, Messner D, Pierce GF, Witkop M, Tunis S; for the coreHEM panel. Core outcome set for gene therapy in haemophilia: Results of the coreHEM multistakeholder project. Haemophilia. 2018;00:1 '6. https://doi.org/10.1111/hae.13504
coreHEM: Developing Comparative Effectiveness Outcomes for Gene Therapy in Hemophilia
coreHEM has produced the first set of guidelines recommending a specific, minimum set of outcomes to include in hemophilia gene therapy clinical trials. This core set will ensure that patient perspectives on critical outcomes are included in pivotal trials, allow fair comparisons between alternative treatments, and allow more accurate assessments of the value of these therapies. The coreHEM project was jointly led by the Green Park Collaborative (GPC), the National Hemophilia Foundation (NHF), and McMaster University.