Ana Pena, PhD,  —

Pregnant women who are hemophilia B carriers have more than three times the risk of bleeding following childbirth than non-carriers, a register-based study done in Sweden has found. In contrast, researchers found no differences in the risk of postpartum bleeding between hemophilia A carriers and non-carriers, or in the…

Updated results from the Alta trial show that a single infusion with the highest dose of SB-525, an investigational gene therapy, yields dose-dependent and durable increases in clotting factor VIII (FVIII). The trial, in adults with severe hemophilia A , found no bleeding episodes up…

Marstacimab, an experimental treatment in clinical testing for hemophilia A and B, was able to restore clotting in hemophilic blood and plasma tested in laboratory assays, according to a study. If proven safe and effective in patients, the investigational therapy could provide an alternative preventive solution to…

Preventive treatment with Hemlibra (emicizumab) is superior to replacement therapy with factor VIII infusions for reducing bleed rates in people with hemophilia A without inhibitors, a pooled analysis of trials shows. The study “Efficacy of emicizumab prophylaxis versus factor VIII prophylaxis for treatment of hemophilia A…

Cell therapies may be a feasible way of achieving a long-term correction of bleeding disorders, preclinical data shared by Sigilon Therapeutics show. A single implant of Sigilon’s engineered cells given to mice enabled durable production of therapeutic levels of blood clotting factors and normalized bleeding time. The technology uses the…

The ongoing Phase 1/2 Alta trial continues to show that a single infusion of SB-525, an investigational gene therapy, leads to stable and clinically relevant improvements in clotting factor VIII and marked reductions in the need for replacement therapy in patients with hemophilia A. This preliminary follow-up data…

U.S. doctors caring for people with hemophilia are more likely to make treatment decisions based on patient preferences than doctors in the U.K., who tend to be more influenced by their colleagues and government policies, according to a study comparing decision-making in both…

A single infusion of AMT-061, an investigational gene therapy for hemophilia B, continues to maintain clinically significant increases in clotting factor IX (FIX) up to six months after its administration, as demonstrated in all three patients enrolled in uniQure…

Bleeding episodes, chronic pain, and the presence of inhibitors in children with hemophilia cause emotional stress for caregivers and increase their burden, according to an European study that recommended early access to support services. Caring for a child with hemophilia, particularly when coping with poor joint health, leads to emotional stress,…

Maintaining high concentrations of Adynovate in the blood of hemophilia A patients decreases the number of bleedings and enables more patients to stop having bleeds, according to the first results of a Phase 3b/4 study by Takeda.