The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to GENV-HEM, GeneVentiv Therapeutics’ investigational gene therapy for hemophilia A and B patients, with or without clotting factor inhibitors. Orphan drug designation is given to treatment candidates that have the potential to be safe and effective…

A single infusion of Hemgenix (etranacogene dezaparvovec-drlb) helped boost levels of factor IX (FIX), the clotting protein missing in hemophilia B, in many patients who already had antibodies against the viral carrier, according to new trial data. The findings suggest the gene therapy may benefit a broader group of…

Generation Bio said it received $110 million in funding to bring its innovative, non-viral gene therapy for lead programs hemophilia A and phenylketonuria into investigational new drug-enabling clinical trials. Gene therapy is an investigational treatment technique that uses genes or genetic material to treat or prevent…

Inadequate access to treatment centers, lack of expert care, and high bleeding rates lead to similar joint complications in hemophilia patients with and without inhibitors, and joint problems in 70% of the people taking part in a recent study in five developing countries. The research, “HAEMOcare:…

Instead of Powerball, I have won the rare bleeding disorder lottery. I have von Willebrand disease and hemophilia B, a less common form of hemophilia that arises when a blood-clotting protein called factor IX is missing or deficient. My diagnoses are rare within a rare community. I often feel like a…

Discovering your child has hemophilia can be devastating. While you will certainly need to make some lifestyle changes moving forward, with the correct treatment and preventative measures, there’s no reason why your child can’t live a happy and healthy life. To help you come to terms with life with hemophilia and…

Preventive therapy, or prophylaxis, in patients ages 50 and older with severe hemophilia type A  and type B was effective at reducing bleeds, including those in target joints, and improving some aspects of their quality of life. That’s according to findings from a post hoc analysis of four…

When dealing with health issues, does when you were born matter? For those living with hemophilia, the answer is a resounding yes. Having personally experienced the challenges of living with bleeding disorders and survived multiple life-threatening situations, I can’t help but reflect on how different my medical journey might…

Hemophilia is a rare blood condition where people do not have the clotting factor which 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…

It’s a struggle to balance life with two disorders. I need to take daily medications for epilepsy while also self-infusing to treat internal bleeds. I recently suffered a horrible epileptic episode. My wife, Cza, and I were walking with our baby, Cittie, when I felt a strong aura warn…