Intellia Therapeutics and Regeneron are planning to begin, by mid-year, the first-in-human trial testing a CRISPR/Cas9-based Factor 9 (F9) gene-editing therapy for people with hemophilia B. The announcement of the planned launch of the Phase 1 clinical trial, in an Intellia financial report, follows the recent approval by…
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Sarcopenia, or loss of muscle mass and strength, may occur in as many as half of adults with severe hemophilia A who experience recurrent joint bleeds, according to a small study in Turkey. In addition, measuring muscle mass in the thighs with ultrasound, which is a noninvasive imaging test…
A real-world study in Spain found that, among hemophilia patients, those with hemophilia A may have have worse joint health — and more joint damage, particularly in the ankles — than those with hemophilia B. Importantly, though, such damage appeared to be less frequent for individuals on regular…
Almost three years of treatment with SerpinPC was associated with a nearly complete resolution of bleeds and a reduction in the number of affected joints among men with severe hemophilia A or B who participated in a Phase 1/2a clinical trial. The…
Women with hemophilia and other bleeding disorders are more likely to experience postpartum hemorrhage and other bleeding problems related to pregnancy and childbirth than those without such diseases, a population-based study from Canada reports. Findings “showed that women with inherited bleeding disorders are at increased odds of [postpartum hemorrhage]…
Individually tailored prophylaxis, or preventive treatment, with Jivi (damoctocog alfa pegol) nearly halved the number of annual bleeding episodes in people with severe hemophilia A who had previously been on another replacement therapy, according to a Phase 4 post-marketing study. Researchers also observed that none of the patients…
Over seven years of follow-up, a single dose of Roctavian (valoctocogene roxaparvovec-rvox) sustainably reduced the yearly bleeding rate in adults with severe hemophilia A by up to 96%. That’s according to an update on a Phase 1/2 clinical trial (NCT02576795), launched in the U.K. in 2015, that’s…
Unexpectedly high demand for the 3,000 international units (IU) vial size of Rebinyn (nonacog beta pegol) has resulted in a temporary shortage of the Novo Nordisk factor IX (FIX) replacement therapy for those with hemophilia B, according to the company. In a statement, Novo Nordisk said…
Progressive joint disease caused by repeat bleeds, a condition known as hemophilic arthropathy, affects people with moderate hemophilia in Nordic countries regardless of age, commonly causing them pain and restricting their mobility, a study found. Middle-age patients, those 41 to 50 years old, felt their overall quality of life…
The development of neutralizing antibodies, or inhibitors, against the clotting factor in replacement therapies administered to people with hemophilia may depend on the type of product used, a recent study has found. In previously untreated people with severe hemophilia A, or those who lack…
