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 People who used the therapy for nearly three years saw regular improvements in their joint health. |
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Monday, November 6, 2017  The BLA was submitted in light of positive data yielded from a phase 2/3 clinical study. |
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Monday, October 30, 2017  The breakthrough status was granted in light of positive results of phase 1/2 clinical study. |
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Friday, October 27, 2017  If effective, the therapy prompts the sustained production of FVIII and enhanced protection from bleeds. |
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Friday, October 20, 2017  UniQure announced plans to “expeditiously advance” its investigational hemophilia B gene therapy AMT-061 into a study in 2018. |
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Monday, September 25, 2017  The company stated subcutaneous injections of the therapy boosted FIX in hemophilia B patients to therapeutically significant levels. |
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Friday, September 8, 2017  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. |
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Thursday, September 7, 2017  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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Wednesday, September 6, 2017  Bayer applied for a Biologics License Application for BAY 94-9027, the company’s long-acting site-specific PEGylated recombinant factor VIII therapy for the treatment of hemophilia A. |
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Tuesday, September 5, 2017  Sangamo Therapeutics, Inc. and Pfizer Inc. recently announced that the first patient received treatment in a phase 1/2 clinical trial designed to evaluate SB-525, the company’s gene therapy program for people with hemophilia A, or factor VIII (FVIII) deficiency. |