Emicizumab trial shows significant improvements in reducing bleeds in people with hemophilia A and inhibitors.
Results of recombinant product on previously untreated patients provide data on inhibitor development.
Significant improvements seen in the patients in the trial, some of whom were followed for one year.
Experimental hemophilia B therapy shows evidence of better bleed control when the trough level is higher.
Recombinant extended half-life therapy stretches infusion intervals and improves quality of life across different ages tested.
Alnylam Pharmaceuticals, Inc., reports positive interim results from a part of its ongoing Phase 1 study with fitusiran in patients with hemophilia A and B with inhibitors.
Gene therapy clinical trials eliminate infusions in some patients and improve quality of life.
Trials of Sangamo's gene therapy called SB-525 show promising results in animals with hemophilia A.
WILATE®, a human-derived von Willebrand factor/coagulation factor VIII therapy, will be used in two upcoming clinical trials slated to launch in December 2016.
Gene editing therapy will be used in clinical trials first in people with hemophilia B, then in those with hemophilia A.
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