Preventive treatment with Hemlibra (emicizumab) appears to be safe and helps keep bleeding under control in babies who have severe hemophilia A. The number of bleeds was “zeroed out” in nearly half of the babies, and most had zero bleeds requiring treatment. That’s according to interim results from…

Two new mutations affecting the production of functional clotting factor VIII were identified in a group of Vietnamese patients with hemophilia A, researchers report. Their findings were described in a letter to the editor, “Mutation characteristic of 103 haemophilia A patients in Vietnam: Identification of novel mutations,”…

Scientists in Taiwan have created a new point-of-care device to diagnose hemophilia type A and determine a person’s blood type using just a small sample of blood. The device “is especially suitable for usage in emergency or natural disasters to provide quantitative testing in rescue and relief operations,” the…

Hemophilia is a rare inherited disease where a person is lacking a certain type of clotting factor. This means that their blood is unable to clot so injuries will result in extensive bleeding. If left untreated, this bleeding can become life-threatening. Here are a few fast facts about this rare…

In people with hemophilia A, certain immune markers in the blood may help predict patient responses to immune tolerance induction (ITI) — a treatment regimen used for individuals who develop inhibitors after factor replacement therapy — according to a new study by researchers in Brazil. Inhibitors, neutralizing antibodies…

The European Medicines Agency (EMA) is asking for a full year’s worth of data from a Phase 3 trial of Roctavian as it considers approving this potential gene therapy for adults with severe hemophilia A, its developer, BioMarin, reported. The request by the regulatory agency for the European…

Hemophilia is a disease where a person blood lacks a factor which enables it to clot. This can lead to severe bleeding which can be life-threatening if left untreated. According to the World Federation of Hemophilia, there are three types of hemophilia: inherited, sporadic, and acquired. MORE: Five…

The U.K. Medicines and Healthcare products Regulatory Agency (MHRA) has approved efanesoctocog alfa — to be sold under the brand name Altuvoct —  as a treatment for moderate or severe hemophilia A in adults and children, ages 2 and older. Approved in the European Union in June…

GreenGene F (beroctocog alfa), a lab-made version of coagulation factor VIII, has been approved by regulators in China for the control and prevention, or prophylaxis, of bleeding episodes associated with hemophilia A, the therapy’s developer, GC Pharma,  announced. GreenGene F will be available in China in the first…

Preliminary results of a Phase 1/2a trial of the hemophilia A experimental treatment BIVV001 show a significant extension of replacement therapy lifetime in the blood, Bioverativ announced. The ongoing, and still recruiting, open-label, multicenter EXTEN-A study (NCT03205163) is evaluating the safety, tolerability, and pharmacokinetics of a single intravenous injection…