Children’s Hospital of Philadelphia (CHOP) has been awarded up to $38.9 million to advance the development of personalized gene‑editing therapies for four groups of rare genetic disorders — one of them hemophilia. The five-year award comes from the Advanced Research Projects Agency for Health (ARPA‑H) under its…
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Although it’s often mistakenly thought of as a male disease, hemophilia affects a substantial number of women and girls, manifesting with symptoms that can include heavy menstrual periods and mental health challenges. That’s according to a new review paper that examined “each stage of the process” for female patients…
Denecimig (Mim8), an investigational antibody therapy being developed by Novo Nordisk, continued to maintain low bleeding rates in children, adolescents, and adults with hemophilia A, with safety findings consistent with earlier studies, regardless of inhibitor status or dosing schedule. That’s according to interim data from FRONTIER4 (NCT05685238), an…
For couples affected by hemophilia who want to avoid passing the inherited bleeding disorder to their children, early genetic testing — done during the in vitro fertilization (IVF) process — may offer a reliable option for family planning, a new study from Italy suggests. That study investigated a procedure…
The Haemophilia Joint Health Score (HJHS), a standard tool used to assess joint health in people with bleeding disorders, including hemophilia, may underestimate joint damage, or haemarthropathy, in women and girls, a study suggests. That’s because the HJHS assesses joint mobility by comparing a person’s joint range of motion…
A new type of gene-editing treatment being developed by researchers in Japan corrected mutations causing severe hemophilia B in mice, restoring production of the missing clotting factor IX (FIX), which causes the rare bleeding disorder. The scientists say their novel nonviral treatment — which does not use an inactivated…
In a small study, children with hemophilia A had distinct changes in certain immune cells and inflammatory signaling molecules compared with healthy children, and the type of preventive treatment they receive may shape those immune responses in different ways, according to the researchers. In lab experiments, macrophages — immune…
Nearly one-third of U.S. hemophilia treatment centers (HTCs) do not involve genetic counselors in patient care or refer patients for genetic counseling, despite widespread recognition among healthcare providers that these specialists offer valuable expertise, a U.S. survey found. Healthcare providers across treatment centers recognized the value of genetic counselors…
Researchers spent years searching for a new treatment option for people with hemophilia A or B before arriving at the therapy that became Hympavzi (marstacimab). Scientists at Pfizer, the company that markets Hympavzi, described the search in a study, “Discovery and optimization of marstacimab, a human monoclonal antibody…
Repeated dosing of the long-lasting factor replacement therapy Altuviiio (efanesoctocog alfa) over a short timeframe may not increase the risk of dangerous clotting events, according to studies in a mouse model of hemophilia A. Blood analysis showed an increase in global coagulation function, which measures the formation and…
