Most people with severe hemophilia A who were given once-weekly treatment with efanesoctocog alfa in the Phase 3 XTEND-1 trial were bleed-free over a year on treatment. The therapy also improved joint and overall physical health and eased pain intensity in the participants compared with previous treatments. Results…

Incidences of bleeding from playing sports are rare among people with hemophilia, and more likely to occur in those with lower levels of clotting factors — proteins that play a key role in promoting blood clotting — a study in the Netherlands has found. More specifically, the study reveals…

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…

The first of my four alarms sounds at 6 a.m. Groggy, I roll over to turn it off, knowing I have 15 minutes until the next one goes off. I am a night owl and often let time slip away, crawling into bed after midnight. The next morning, I pay…

Officials of the U.S. Food and Drug Administration and Alnylam Pharmaceuticals have agreed on safety measures and a “risk mitigation strategy” to allow the resumption of clinical studies of fitusiran, the company said in a recent press release. All ongoing trials of fitusiran had been suspended after the Sept. 7 death…

An ultrasound-mediated, non-viral gene therapy safely and effectively increased the levels of factor VIII (FVIII) — the missing clotting factor in hemophilia A — and lessened bleeding in a mouse model of the disease, a study shows. The delivery of a modified, improved version of the disease-associated F8 gene…

Aptevo Therapeutics announced the dosing of a first patient in a Phase 4 study aiming to extend use of its prophylactic (preventive) treatment Ixinity (coagulation factor IX [recombinant]) to children with hemophilia B  younger than 12 years old. “Initiation of the clinical study of Ixinity in…

I recently attended a national conference for women with hemophilia. Common themes emerged as I listened to women’s stories of life with bleeding disorders. The number of women dismissed by top medical professionals working for hemophilia treatment centers (HTCs) was alarming. I wondered why this is happening. Dominant…

Hemophilia is a rare disorder where the person is unable to stop bleeding due to a lack of certain blood clotting factors. It’s a genetic disease that’s passed down through the X chromosome. Almost all people with hemophilia are male, however, women can be carriers of the disease. MORE: Explaining hemophilia carriers.

Severe hemophilia B patients receiving prophylactic, or preventive treatment with factor IX face a significant financial and personal burden, according to data from two U.S. population-based medical databases. These findings were reported in a study, “Clinical, humanistic, and economic burden of severe hemophilia B in the United States:…