Genentech recently reported results from the phase III HAVEN 3 study, which showed that the prophylactic use of HEMLIBRA® led to significantly reduced bleeds in adult and adolescent patients with h
Alnylam is developing the therapy to treat patients with hemophilia A and B, both with and without inhibitors.
People who used the therapy for nearly three years saw regular improvements in their joint health.
The BLA was submitted in light of positive data yielded from a phase 2/3 clinical study.
The breakthrough status was granted in light of positive results of phase 1/2 clinical study.
If effective, the therapy prompts the sustained production of FVIII and enhanced protection from bleeds.
UniQure announced plans to “expeditiously advance” its investigational hemophilia B gene therapy AMT-061 into a study in 2018.
The company stated subcutaneous injections of the therapy boosted FIX in hemophilia B patients to therapeutically significant levels.
The U.S. Food and Drug Administration has accepted the Biologics License Application (BLA) and granted priority review for emicizumab, as a once-weekly treatment for adults, adolescents and children with hemophilia A with factor VIII inhibitors.
Alnylam Pharmaceuticals, Inc. has reported on the death of a hemophilia A patient who had been participating in a Phase 2 study for the company’s investigational therapy fitusiran.
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