Bioverativ will present some of its most recent therapeutic advancements regarding hemophilia and other blood diseases at the 60th Annual Meeting of the American Society of Hematology (ASH) in San Diego, California, Dec. 1-4. The company, which is part of Sanofi, has focused on the development and commercialization of innovative…
News
Aptevo Therapeutics provided an update on Ixinity, its hemophilia B therapy, that includes developing a larger 3,000 IU vial assay for patient convenience. The company also plans to initiate a Phase 4 clinical trial in a pediatric patients, and to apply for Ixinity’s approval outside of the United States.
#NORDSummit – Major Issues on Table for Rare Disease Patients in US as Midterm Elections Approach
With the U.S. midterm elections now less than two weeks away, patient advocacy groups are solidly focused on a range of hot-button issues, from the Orphan Drug Tax Credit and affordable health insurance to future funding for rare disease research. Yet “whether Democrats take over the House or Senate,…
Bayer’s rFVIII Replacement Therapy Jivi Approved in Canada for Hemophilia A Patients 12 and Older
Hemophilia A patients age 12 and older who live in Canada now may receive prophylactic (preventive) treatment with Bayer’s antihemophilic factor Jivi. Health Canada approved Jivi as a treatment to prevent or reduce the frequency of bleeding episodes in patients age 12 or older who have received…
Re-administering a viral vector-based gene therapy for hemophilia A was shown to be safe and to achieve long-term improvement in a dog model of the disease — even after neutralizing antibodies were evident following the initial treatment. The study, “An observational study from long-term AAV re-administration in…
Students Chosen for “Hemophilia: The Musical” Production Announced by BioMarin and Believe Limited
BioMarin Pharmaceutical Inc. and Believe Limited have announced the students chosen to participate in the theatrical production of “Hemophilia: The Musical,” a program that aims to empower the bleeding community through performance while raising public awareness of the connection between arts and wellness. The 25 high school students hail from…
Octapharma USA Sponsors Retreat for Women With Bleeding Disorders and Screens ‘Bombardier Blood’
There are still too many women with bleeding disorders who, despite symptoms, are unaware of their disease. That circumstance will be a major topic Oct. 19-21 at The Fourth Annual Female Factor Retreat, hosted by the Hemophilia Foundation of Northern California (HFNC). Other topics at the educational retreat, to…
Total joint replacement (TJR) is more common in severe hemophilia patients requiring more frequent treatment, according to a population-based study in Taiwan. The findings also revealed that the incidence of TJR does not differ between hemophilia A and B. The study, “Comparison of Total Joint…
Single or combination therapy with bypassing agents used for hemophilia A patients with inhibitors can be suitable treatment options for children with hemophilia B with factor IX inhibitors as well, a case series suggests. The report, “Alternative treatment options…
The U.S. Food and Drug administration (FDA) has approved Genentech’s Hemlibra (emicizumab-kxwh) for routine prophylactic (preventive) treatment of patients with hemophilia A without factor VIII inhibitors. Hemlibra is now the sole available prophylactic treatment for patients with hemophilia A with and without factor VIII inhibitors that can…
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