Orlando skyline at night, Photo by Bill Dickinson.

The World Federation of Hemophilia (WFH) 2016 World Congress took place July 24-28 in Orlando, Florida.

Drug can prevent bleeding in hemophilia A and B

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Structure of RNA

Results from an ongoing phase 1 study suggest fitusiran, a small interfering RNA therapeutic targeting antithrombin (AT), can restore hemostasis and prevent bleeding in patients with hemophilia A or B, with or without inhibitors. In patients with inhibitors, fitusiran exhibited preliminary evidence of reduced bleeding. In patients without inhibitors, fitusiran reduced the... [Read Article]

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Gene therapy reduces need for FIX prophylaxis

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DNA helices
Image courtesy of NIGMS

The gene therapy AMT-060 can reduce the need for factor IX (FIX) prophylaxis in patients with severe hemophilia B, results of a phase 1/2 study suggest. All of the patients treated in the low-dose cohort of this study have had sustained improvements in their disease phenotype and continue... [Read Article]

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Gene therapy shows promise for severe hemophilia A

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DNA helix
Image by Spencer Phillips

An investigational gene therapy can safely reduce bleeding in patients with severe hemophilia A, a phase 1/2 study suggests. The therapy is BMN 270, a recombinant adeno-associated virus (AAV) vector coding for human coagulation factor VIII (FVIII). Six of the 7 patients treated with the highest dose of... [Read Article]

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mAb could change treatment of hemophilia A, doc says

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Monoclonal antibodies
Photo by Linda Bartlett

Emicizumab, a bispecific monoclonal antibody (mAb), could potentially change the treatment paradigm of hemophilia A, according to a speaker at the World Federation of Hemophilia 2016 World Congress. Results of a phase 1/2 study suggest that emicizumab can be safe and effective as once-weekly prophylaxis in patients with... [Read Article]

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Patients can safely receive less FVIII, study suggests

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Antihemophilic factor

It may be possible for hemophilia patients to receive less factor VIII (FVIII) without increasing their risk of bleeding, according to a study presented at the World Federation of Hemophilia 2016 World Congress. The study showed that hemophilia A patients who received prophylactic FVIII from the home infusion provider Option Care received 6... [Read Article]

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Therapy seems safe, effective in kids with hemophilia

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Vial of Adynovate
Photo courtesy of Baxalta

Results of a phase 3 study suggest the full-length recombinant factor VIII therapy Adynovate (BAX 855) can be safe and effective as twice-weekly prophylaxis and to control bleeding in children with hemophilia A. None of the patients in this study developed inhibitory antibodies, and there were... [Read Article]

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