News

Scores of virtual events are afoot around the world to mark Rare Disease Day 2021 on Feb. 28. The activities are focused on heightening awareness about rare diseases and the hundreds of millions of individuals they are thought to affect. Patients, caregivers, and advocates worldwide will sport denim ribbons…

A single dose of the experimental gene therapy SPK-8016 showed promising safety and efficacy in lowering the frequency of bleeds and the need for infusions in men with severe hemophilia A. These preliminary findings from an ongoing Phase 1/2 trial (NCT03734588) were presented by Spencer Sullivan, MD,…

The investigational medication marzeptacog alfa activated (MarzAA) showed promise at stopping bleeding in animal models of hemophilia. The findings were presented in two posters at the Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD) by researchers from Catalyst Biosciences, which is developing MarzAA. MarzAA is…

Kovaltry (octocog alfa) is effective and safe as a preventive treatment for children with hemophilia A, a European real-world study reports. The study, “Real-world evidence on Kovaltry (81-8973) in children with moderate or severe hemophilia A in Europe: a…

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…