The Boston-based Institute for Clinical and Economic Review (ICER) says a “draft scoping document” outlining its planned comparative review of emicizumab‘s clinical effectiveness and value in treating hemophilia A will be available until Sept. 29. ICER’s report will be subject to a public deliberation in March 2018 by the…

Life with hemophilia can be challenging but there are ways that you can manage the disease to ensure your child leads as normal a life as possible. To help you navigate your child’s illness and help prevent bleeds, we’ve put together a list of tips. MORE: How to prepare for your child’s…

Results of the first group of patients with hemophilia B treated with Catalyst Biosciences’ lead candidate CB 2679d in a Phase 1/2 clinical trial demonstrated its improved stability and activity compared to Pfizer’s BeneFix standard-of-care replacement therapy. CB 2679d (also known as ISU304), was designed to be the…

Hemophilia is a rare blood condition where people do not have the clotting factor that enables their blood to clot when bleeding. It’s an inherited disease that’s usually passed from mother to son. It’s also a disease that’s been prevalent in European royal families. MORE: A brief history of hemophilia…

Dimension Therapeutics may soon become a wholly owned subsidiary of Regenxbio, following the terms of an agreement between the two companies. The merger agreement, if finalized, will add Dimension’s investigational drug candidates being developed to Regenxbio’s pipeline, including DTX201 for the treatment of hemophilia A. DTX201 was being developed…

This children’s video from Centers for Disease Control and Prevention focuses on how to tell your friends that you have hemophilia. MORE: How kids can play safe with hemophilia Billy, a young boy with hemophilia, answers some of the questions that his friends ask about the disease. He…

A new online service allows patients with bleeding disorders like hemophilia to share important information about their treatment and bleeds with their healthcare team in real time. MORE: Five tips for living healthily with hemophilia The HemaGo XChange portal was created by Novo Nordisk A/S as an update to their existing website…

In this video shared by Colleen, learn about a mobile app from Pfizer that helps hemophilia patients track bleeds, infusions and factor supply. It also allows those living with the disease to track their daily physical activities, steps and heart rate to help…

Alnylam Pharmaceuticals has suspended patient dosing in all ongoing studies of its RNA therapy fitusiran to treat hemophilia A or B, after a patient died of swelling in the brain in a Phase 2 open-label extension trial. The company will work with the regulatory agencies in reviewing the cause of the…

Bayer has filled a Biologics License Application (BLA) with the U.S. Food and Drug Administration (FDA) requesting marketing approval for its long-acting human Factor VIII therapy, BAY94-9027, to treat hemophilia A. The therapy is designed to allow for dosing at intervals as long as once every seven days. The…