Should regulatory authorities agree with favorable results from Genentech’s ongoing Phase 3 studies of Hemlibra (emicizumab-kxwh),  hemophilia A patients may have a treatment they can use regardless of inhibitor status, with a choice of three dosing schedules that might better match their lifestyle and preferences. “The ultimate goal for…

Some of my most enjoyable moments are guy times with my boys. We laugh, we attempt to keep the house in order, and we succeed, despite ourselves. My sons, who have my blood running through their veins, remind me that life will be OK; that any problem can be solved,…

Data from ongoing Phase 3 clinical trials show that Genentech’s Hemlibra (emicizumab-kxwh) prophylaxis — or preventive treatment — greatly outperforms current standard-of-care therapies, leading to significant reductions in the number of bleeds in hemophilia A patients regardless of inhibitor status. Promising results from these pivotal studies may lead…

Patient advocacy groups in five states will focus on the challenges affecting patients with blood disorders like hemophilia with the support in part of grants from the Local Empowerment for Advocacy Development (LEAD) program, sponsored by CSL Behring. The five groups are the Bleeding Disorders Foundation of Washington (BDFW), the …

Chronic joint inflammation lowers the health-related quality of life in people with severe hemophilia, a European study reports, and urges physicians to be aware of this association in treating patients. The research, “The impact of severe haemophilia and the presence of target joints on health-related quality-of-life,” appeared in…

The Arizona Hemophilia Association (AHA) is inviting camp professionals in the bleeding disorders and hemophilia communities to participate in the National Camp Exchange (NCE) program. Applications are open until May 30 for 2018 summer programs. NCE is set up to give those who plan, organize, and operate…

My mighty warrior Caeleb came home from school last week with a question. “Mom,” he asked, “my knee is really messed up, isn’t it?” I couldn’t see an injury, and so I asked him what he meant by “messed up.” “I can’t run really fast like everyone else in gym class,”…

A Phase 1/2 trial (NCT03520712) testing BioMarin Pharmaceutical’s investigational gene therapy valoctocogene roxaparvovec in severe hemophilia A patients with pre-existing AAV5 antibodies has dosed its first participant. “Administration of valoctocogene roxaparvovec to this first patient seropositive for the AAV5 capsid is an important next step in our plan to…

With my oldest son, who is now 21, I never worried about limitations. He played soccer for a season, and that was the extent of his athletic pursuits. He just did not like sports and to this day, he does not understand the basics of a football game,…

In recognition of World Hemophilia Day 2018, the biopharmaceutical company Bioverativ is sharing the stories of people with hemophilia in the developing world to highlight the life-changing potential of adequate treatment. In honor of this year’s theme, “Sharing Knowledge Makes Us Stronger,” Bioverativ will collaborate with members of the hemophilia community…