News

BioMarin Pharmaceutical Inc. is partnering with digital content agency Believe Limited to produce a three-day musical workshop for young members of the bleeding disorders community. The “Breaking Through!” musical theater intensive, the first program of its kind, will be held Nov. 9-12 in New York City, and aims at teaching…

A panel of multidisciplinary experts have developed recommendations for applying best practice guidelines in the planning and managing of surgery in hemophilia patients. The guidelines were outlined in the consensus review, “Recommendations on multidisciplinary management of elective surgery in people with haemophilia,” published in the journal…

Although moderate to severe hemophilia patients are generally protected against the development of cardiovascular disease, cardiovascular-related events can still occur in this patient population, a study reports. These findings suggest that efforts should be made to prevent cardiovascular disease in hemophilia patients who have risk factors associated with the…

Key leaders in hemophilia therapy development, representing large pharmaceutical and biotech firms as well as academia and contract research organizations, will gather at the Hemophilia Drug Development conference in Boston on Aug. 14 through 16. The meeting is designed to be an industry-dedicated networking and discussion forum, and many of…

UniQure has enrolled the first patient in its Phase 3 HOPE-B single-dose trial investigating the effectiveness and safety of AMT-061 in treating people with severe or moderately severe hemophilia B. AMT-061 uses a viral vector to deliver the gene for a mutated clotting factor IX (FIX), which is also known as…

Octapharma announced the launch of the PREVAIL clinical study (NCT03344003), which will investigate the use of Wilate for treatment of hemophilia A patients in Canada who recently have developed inhibitors to a factor VIII (FVIII) concentrate, or have had long-term inhibitors against FVIII. Hemophilia A is a…

Treatment with CB 2679d increased factor IX activity levels in the blood, bringing them closer to normal levels in the newest group of patients with severe hemophilia B in the Phase 1/2 FIX clinical trial, Catalyst Biosciences announced in a press release. Catalyst, the therapy’s maker, believes that the next-generation factor…

Oxidation of clotting protein factor VIII (FVIII) increased the immune response to the replacement therapy in a mouse model of severe hemophilia A, a study found. Conducted by researchers in France, the study, “Oxidation of factor VIII increases its immunogenicity in mice with severe hemophilia…

Hemophilia patients who develop inhibitors against treatment face markedly higher healthcare costs — about three times those of other patients — and experience poorer quality of life, according to a team of scientists in Italy. Alternatives are available and should be considered for these patients, they write. The study, titled…

Express Scripts has teamed up with PinnacleCare to include in its new Rare Conditions Care Value (RCCV) program a service that provides patients with rare diseases like hemophilia a second opinion from a specialist care provider. The initiative, simply called Second Opinion, is designed to give patients an additional…