https://www.selleckchem.com/pr....oducts/gsk1120212-jt
Early-stage gene therapy (GT) clinical trials are demonstrating exciting results for persons with haemophilia (PWH), with the first products possibly licenced over the next few years for haemophilia A and B. These new treatments offer the possibility of a one-off approach to the treatment of haemophilia, with demonstrated increases in factor level expression and substantial reductions in both bleeds and factor utilization. However, clinical trial participants have demonstrated variable expression in factor levels, including
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