Marisa Wexler, MS,  senior science writer—

The vast majority of women who have a bleeding disorder or are hemophilia carriers experience noticeable symptoms, such as bruising or heavy periods, a study based on survey results reported. These women also often mentioned encountering healthcare professionals who were dismissive or lacking in empathy. “Many of the experiences…

People with hemophilia may be less likely to develop cardiovascular disease (CVD) — disorders of the heart and blood vessels — according to a study from the U.K. and the Netherlands. “A lower-than-predicted [cardiovascular disease] incidence was found, supporting the theory that hemophilia protects against CVD,” the researchers wrote.

Nearly all men with moderate-to-severe hemophilia B given the experimental gene therapy EtranaDez (etranacogene dezaparvovec) in the HOPE-B trial have stopped using prophylactic therapies, full study results show. Trial data also indicated that EtranaDez, formerly known as AMT-061, is effective in people with antibodies against the viral vector…

More than 80% of the men with severe hemophilia A treated with the investigational gene therapy Roctavian (valoctocogene roxaparvovec) in the Phase 3 GENEr8-1 trial remained bleed-free two years later, and nearly all were off standard preventive therapies. These findings were among new data presented at the recent 15th Annual…

Obizur (susoctocog alfa), a pig-derived replacement therapy, can be used to prevent bleeding in people with acquired hemophilia A who need to undergo surgery, a new case report highlights. The report, “Successful perioperative prophylaxis with susoctocog alfa in a patient with acquired haemophilia A: A…

The European Medicines Agency (EMA) has approved an accelerated assessment request for etranacogene dezaparvovec, an experimental gene therapy for hemophilia B. The decision means that, once an application is submitted seeking approval for marketing authorization of etranacogene dezaparvovec, it will be reviewed more quickly than normal — which…

Monthly treatment with the investigational medication fitusiran can reduce bleeding in people with hemophilia A and B, with or without inhibitors, according to data from two Phase 3 clinical trials. “We are encouraged by the data from these initial Phase 3 studies demonstrating fitusiran’s potential as a new…

Amarna Therapeutics has secured €5 million (about $5.6 million) in new funding, which it plans to use for developing its lead product, AMA005, an investigational gene therapy that aims to restore blood clotting in people with hemophilia B. The goal is to advance the company’s hemophilia B program…

In the first clinical trial of the experimental therapy SIG-001 for hemophilia A, one patient developed scarring in the spheres used to deliver the therapy that rendered the treatment ineffective, its developer, Sigilon Therapeutics, announced. The Phase 1/2 trial was put on hold by the U.S. Food and Drug…

Preventive treatment with Hemlibra (emicizumab) is safe and can effectively prevent bleeds in people with mild or moderate hemophilia A, according to an interim analysis of data from the HAVEN 6 clinical trial. Results will be presented at the annual meeting of the American Society of Hematology (ASH)…