News

Implanting bioscaffolds — 3D biological scaffolds made up of collagen and other molecules — carrying transformed liver cells into rats successfully triggered the production of factor VIII (FVIII), the clotting protein missing in people with hemophilia A. According to researchers, these bioscaffolds may be a therapeutic alternative to FVIII…

The first documented case of a previously untreated hemophilia B patient developing inhibitors against the extended half-life therapy Alprolix was described in a report from Canada. The report also shows how combining routine administration of Alprolix with medications that suppress the immune system can be used to stop…

Global Genes has partnered with the Rare Disease Diversity Coalition (RDDC) to advance health equity for rare disease patients and caregivers in underrepresented communities of color. “For rare disease patients, there are many challenges — and for people of color with a rare disease, these challenges are compounded…

Eptacog beta was safe and 100% effective at preventing bleeds during and after minor surgeries in hemophilia A patients with inhibitors, according to data from a Phase 3 trial. The treatment’s efficacy at managing bleeds dropped with major surgeries, with post-operative success seen in 66.7% of evaluated cases. Overall, these…

Long-term prophylactic (preventive) treatment with a fixed dose of Esperoct (turoctocog alfa pegol) — approved in the U.S. and the EU in 2019 — was effective in preventing and stopping bleeding in people with severe hemophilia A, according to data from two Phase 3 clinical trials. Findings from…

Poseida Therapeutics and Takeda are working together to develop non-viral gene therapies for hemophilia and other genetic diseases. “Together with Takeda, we look forward to developing potential cures for a number of genetic diseases with high unmet need,” Eric Ostertag, MD, PhD, Poseida’s CEO, said in a press…

The Rare Disease Diversity Coalition (RDDC) awarded $600,000 in grants to ease the disparities faced by rare disease patients of color. These Impact Rare Disease Solution grants will go five RDDC steering committee working groups, which aim to identify problems for rare disease communities and advocate for solutions. The five…

The World Federation of Hemophilia (WFH) is commemorating having successfully distributed, since 1996, over one billion international units (IUs) of factor and non-factor replacement therapies for people with inherited bleeding disorders, such as hemophilia, through its Humanitarian Aid Program. Donations from the program have improved the quality…

A newly designed platform based on the principle of “reverse vaccination” could be used to prevent the development of an immune response that blocks the effectiveness of treatments for hemophilia and other disorders. “For nearly a third of patients with hemophilia A … their own immune system is…

The Hemophilia Federation of America (HFA) and the National Hemophilia Foundation (NHF) jointly launched the “Together Project,” in which the two organizations will work together to address issues affecting the bleeding disorders community. Most of the partnership’s efforts will center on written content creation and visual designs, which…