News

A daily infusion of low-dose factor VIII (FVIII) improves trough levels in patients with severe hemophilia A — compared to infusions performed every other day — without increasing factor VIII consumption, or the amount of medication a patient must take, researchers have found. Trough levels are the lowest concentrations of a…

The hemophilia A treatment Nuwiq (simoctocog alfa) is safe and effective for patients with severe disease, both when used on demand or as a preventative treatment, researchers determined in a recent report. The researchers, who analyzed available data on Nuwuq, detailed their finding in the article, “Efficacy and…

Most young people with moderate or severe hemophilia A and B in France receive long-term prophylactic (preventive) treatment — an approach linked to a lower rate of bleeding episodes than among patients treated on demand. The study, “Choice of factor VIII/IX regimen in adolescents and young adults with…

A study presented at American Society of Hematology’s (ASH) annual meeting and exposition shows that Adynovate is effective for the prevention and treatment of severe pediatric Hemophilia A (HA). Eric Mullins, MD and his colleagues from the Cancer and Blood Diseases Institute at Cincinnati Children’s Hospital Medical Center presented their work…

Damaged joints and bones in mice with hemophilia benefit from extended activity of coagulation factor IX, research shows. The study, “Abnormal joint and bone wound healing in hemophilia mice is improved by extending factor IX activity after hemarthrosis,” was published in the journal Blood. Patients with hemophilia often have…

Positive mental attitudes and encouragement from family and friends are key reasons young hemophiliacs participate in sports that could increase their risk of bleeding, a study says. The research showed that psychosocial factors play a larger role in their taking part in discouraged physical activity than in their accepting other…

Sangamo Therapeutics recently presented what it characterized as key improvements to its technology platform for engineering highly specific zinc finger nucleases (ZFNs), which are used for targeted editing of the human genome. This technique has broad applicability, namely for the treatment of hemophilia B and other disorders. The…

Xenetic Biociences has received a $3 million milestone payment from its partner Shire for advancing the Phase 1/2 clinical trial of PSA-recombinant SHP656, a factor VIII protein, evaluating the drug’s effectiveness as a long-acting therapy for hemophilia A. Shire’s main objective behind the agreement is to develop a novel FVIII…

A Sangamo Therapeutics‘ gene therapy program to potentially treat hemophilia A, called SB-525, has been cleared by the U.S. Food and Drug Administration (FDA) for clinical testing in adult patients. The company announced that the FDA approved its Investigational New Drug (IND) application for SB-525, a necessary first step…

The U.S. Food and Drug Administration (FDA) has approved Shire’s Adynovate for the treatment of pediatric patients under 12 years of age with hemophilia A. Adynovate also was approved for use by both adults and children undergoing surgical procedures, to control bleeding episodes. Adynovate is a recombinant Factor…