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,…
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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…
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…
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…
Half a year has gone by since disgraced pharma executive Martin Shkreli was sentenced to seven years in federal prison for securities and wire fraud while heading San Diego-based Retrophin. As founder and CEO of another company (then known as Turing Pharmaceuticals), in late 2015 Shkreli bought the rights to…
More than 700 medical experts, pharmaceutical executives, patient advocates, and others are expected to converge on Washington, D.C., next month for the 2018 NORD Rare Diseases & Orphan Products Breakthrough Summit. The Oct. 15-16 event, sponsored by the National Organization for Rare Diseases (NORD), takes place at the…
