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…
A crowdfunding campaign aims to raise $45,000 to support “Rare,” a documentary film featuring the struggles and achievements of people living with rare diseases and their families. Sweis Entertainment and Digital Cave Media launched the campaign — allowing filmmakers to finish producing and to release the documentary — on Kickstarter.
Provisions of a new law that went into effect on Oct. 1 allow families affected by hemophilia and other bleeding disorders who are under Medicare to better access skilled nursing facilities (SNFs), the National Hemophilia Foundation (NHF) announced. This law, known as the Hemophilia SNF Access Act, should…
Differences in sensory detection and pain thresholds were found in hemophilia patients taking prophylactics, compared to healthy individuals, and these differences did not change over the course of a one-year follow-up study. These findings indicate that “under prophylactic treatment, the existing difference in the pain profile between [people with…
People with hemophilia are nearly four times more likely to experience sexual problems than are individuals without bleeding disorders, according to the results of a new international survey. The survey found that problems with sexual intimacy in patients with hemophilia were associated with older age, experiencing acute or chronic pain…
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