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…

Boxes, boxes, and more boxes! We can’t seem to find a way to sift through the endless sea of those irritating but necessary cardboard contraptions containing pieces of our lives. We know that we cannot stop piecing together our lives in another place. Moving forward appears to be the best…

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…

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…

Since every hemophilia A patient responds differently to therapies, researchers are working to develop personalized preventive (prophylaxis) strategies with Nuwiq (antihemophilic factor [recombinant].) Octapharma, the treatment’s manufacturer, presented data showing that an individual’s pharmacokinetic (PK) profile (how the therapy works once inside the body) can be used to possibly…

My son gets off the bus. I look at his face, hoping to see an indication of how his first camp experience went. Did he have a good time? What kind of life lessons did he learn being away from home for a whole week? I looked for answers…

Social media. You either love it or hate it. I must confess that I am guilty of wasting time on Facebook and Twitter, but I have used these outlets as a means of support on more than a few occasions. In the bleeding disorder community, several Facebook groups…