News

Finding treatments and potential cures for rare diseases is crucial, but so is the quality of patients’ lives — a rather nebulous term that means different things to different people. “Recently, there’s been much more of a focus on Quality of Life (QoL) issues, real-world evidence and patient-reported outcomes,” said…

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…

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 National Organization for Rare Disorders (NORD) will celebrate the 35th anniversary of both the 1983 Orphan Drug Act and NORD’s founding at a dinner tonight in Washington, D.C. The 2018 Rare Impact Awards, to be held at the Andrew W. Mellon Auditorium, will be webcast via Facebook for those…

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…

The European Union isn’t doing enough to protect the 30 million or so people with rare diseases who live in its 28 member countries, officials meeting last week in Vienna said. More than 900 people from 58 nations attended the 9th European Conference on Rare Diseases & Orphan Drugs (ECRD), held…

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…

The National Heart, Lung and Blood Institute (NHLBI) is hosting a State of the Science Workshop on May 15-16, 2018, in Bethesda, Maryland, to include input from the hemophilia community into a coordinated strategy for future basic, translational, and clinical research. Although preregistration is closed, on-site registration…