Clinical testing: Previously tested in a Phase 1/2 clinical trial; Phase 3 clinical trial underwayHow does SPK-8011 work in hemophilia?Hemophilia A is caused by mutations in the F8 gene, which provides instructions for making factor VIII (FVIII), a clotting protein found in the bloodstream. ...
研究结果显示,dirloctocogene samoparvovec给药后7年的耐受性良好,FVIII持久表达,以及具有临床意义的ABR和AIR减少。 参考来源:Stacy E. Croteau, et al. Long-Term Follow-Up of Participants in the Phase I/II Trial of Dirlocto...
This Phase 1/2 study is an open-label, non-randomized, dose-escalation study of SPK-8011 with a starting dose of 5x10 11 vg/kg. The study is evaluating the safety, tolerability and efficacy of a single intravenous infusion of SPK-8011 in up to 18 adult men with hemophilia A (...
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