News

While progress was made last year on newborn screening and other policy issues critical to rare disease patients, a “State Report Card” argues that many concerns — notably out-of-pocket costs for prescription medicines and access to affordable comprehensive care — still need attention. Those were the findings of the…

Immunoadsorption, or the selective removal of antibodies from the blood, might prove useful as an add-on treatment for patients with acquired hemophilia, according to a recent study. If confirmed, this finding could lead to better treatment for life-threatening bleeds, delayed treatment responses, and adverse responses to immunosuppressive therapy among…

Severe hemophilia A patients using Esperoct (turoctocog alfa pegol) as a preventive treatment do well, with fewer bleeds or breakthrough bleeds, lower costs, and better life quality compared with other extended half-life (EHL) therapies, a modeling analysis suggested. The…

Although hemophilia treatments are generally effective, women and girls with this disease have specific medical challenges, such as excessive menstrual bleeding, that must be addressed for better clinical care. A study evaluated the symptoms, bleeding management, and clinical outcomes related to menstruation, childbirth, dental procedures, surgeries, and other bleeding events among female…

The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected to pose little risk to the rare disease community, including to patients with compromised immune systems or those participating in gene therapy studies. That was the message of a recent…

CSL Behring has issued a voluntary, pharmacy level recall covering one batch of Mononine, its long-standing treatment for preventing and controlling bleedings in people with hemophilia B that is soon to be discontinued. According to the company’s statement, the recall is a precautionary measure due to…

Rixubis safely and effectively prevents and manages bleeding in children and adults with hemophilia B, a real-world study from South Korea reports. The study, “Safety and effectiveness of Rixubis in patients with hemophilia B: a real-world, prospective, postmarketing surveillance study in South Korea,” was published in the journal Blood…

Caring for a loved one with a rare disease, especially during these uncertain times, demands significant time, attention, patience, and dedication. To help meet that need, the National Organization for Rare Disorders (NORD)’s Rare Caregiver Respite Program may be a helpful resource. The program seeks to give a well-deserved…

People with severe hemophilia B are likely to develop hemophilic arthropathy — a painful and degenerative joint disease caused by recurrent bleedings — despite the lower bleeding frequency relative to hemophilia A, a natural history study shows. The data also highlights that prophylactic (preventive) treatment guidelines are not being…

Eloctate can induce immune tolerance in people with hemophilia A more quickly than other regimens, a study suggests. The study, “Real‐world data of immune tolerance induction using recombinant factor VIII Fc fusion protein in patients with severe haemophilia A with inhibitors at high risk for…