Women and girls with hemophilia continue to face delayed diagnoses, unequal access to treatment, and underrepresentation in clinical trials, even though many experience significant bleeding, according to a new analysis. Researchers described these interconnected problems as the “XX gap” in hemophilia care. Closing this gap, they said, requires moving…
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Long-term treatment with Hemlibra (emicizumab) provided sustained protection against bleeding for up to five years in people with hemophilia A, with no new safety concerns identified, according to a large real-world study from Canada. The study, which analyzed data from 710 people with hemophilia A, found that more…
Recognizing acquired hemophilia A (AHA) early and ordering simple blood clotting tests in older patients with unexplained bruising or anemia — a low number of red blood cells — may help shorten diagnostic delays, according to a Singapore study in which the median time to diagnosis was four days,…
Once-weekly infusions of Altuvoct (efanesoctocog alfa) — a long-lasting hemophilia treatment marketed in the U.S. as Altuviiio — reduced bleeding in adults and children with hemophilia A for whom preventive treatment was not sufficiently effective. That’s the key finding of a new French real-world study that tracked…
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…
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…
