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…
Creative expression within the bleeding disorders community will be on display for the thousands expected to attend the National Hemophilia Foundation’s (NHF) Annual Bleeding Disorders Conference next month in Orlando, Florida. Backed by BioMatrix Specialty Pharmacy, the exhibit, called Blood Work 2018, will showcase interactive gallery submissions from around…
Barry Haarde was a tough man to stop. Despite a decades-long battle with HIV and a near-death brush with hepatitis C, he made history in 2012 as the first person with hemophilia to bike across the country. In fact, he spent the last six years of his life bicycling…
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