News

The International Society on Thrombosis and Hemostasis (ISTH) created a Bleeding Assessment Tool (BAT) that is useful to identify and assess disease severity in people with hemophilia, both  newly diagnosed patients and those who had a prior diagnosis, a study found. The study, “Application of the ISTH bleeding score in hemophilia,”…

Repetitive movements, fitness level, and prolonged and competitive physical activity are among the factors that raise bleeding risks for people with hemophilia taking part in such activities, a study based on a survey of physical therapists in the U.S. reports. Duration of training and safety equipment were seen as modifiable factors,…

Sigilon Therapeutics and the Massachusetts Institute of Technology will work together to develop programmable cell therapeutics for the treatment of hemophilia, lysosomal storage diseases, and other serious chronic illnesses. The collaboration will combine Sigilon’s proprietary cell technology with synthetic biology approaches developed by Ron Weiss, PhD, a…

Combining the bypassing agents Novoseven and Feiba, with extended time between doses, could be a successful and cost-effective protocol to manage bleeding episodes in hemophilia A patients with inhibitors, according to a retrospective study. The study, “Combination of Novoseven and Feiba in hemophiliac patients with inhibitors,” was published in…

Past infection with the hepatitis C virus (HCV) is the major risk factor for hepatocellular carcinoma (HCC), the most common type of liver cancer, a large study analyzing more than 18,000 U.S. inpatients with hemophilia reported.

A single dose of BIVV001 significantly increased levels of factor VIII for a week in the blood of people with severe hemophilia A, according to early results of a Phase 1/2a study. The treatment increased the half-life of factor VIII to 44 hours and showed no side effects or development…

Long-term treatment with Novoeight (turoctocog alfa) is safe and effective at preventing bleeding episodes in patients with hemophilia A of all ages who had already received prior treatment, a Phase 3b extension trial shows. The study, “Long‐term safety and efficacy of turoctocog alfa in prophylaxis and treatment…

Sangamo Therapeutics has dosed the first patient in a Phase 1/2 clinical trial testing its investigational genome-editing therapy, SB-FIX, for patients with severe hemophilia B. The Georgetown University and MedStar Georgetown University Hospital are conducting the study, which is still recruiting participants in nine sites across the United…

Catalyst Biosciences recently announced updates on two of its lead compounds — Factor VIIa (FVIIa) marzeptacog alfa (activated) (MarzAA) and Factor IX (FIX) dalcinonacog alfa (DalcA) — for treatment of hemophilia A or B with inhibitors, and for hemophilia B. DalcA is a potent recombinant (artificial) Factor IX prophylactic therapy designed to prevent acute bleeding…

Cash-strapped governments across the 28-member European Union are struggling to control runaway healthcare expenditures — at exactly the same time as the promise of new but expensive therapies to treat rare diseases has never been greater. That’s the paradox faced by pharmaceutical companies as well as patient advocacy groups in…