I was at the Coalition for Hemophilia B’s Annual Symposium this month in Orlando, Florida — surrounded by my community, sharing meals and meaningful conversations — when it happened. No bump. No injury. No accident. I was literally just standing in the elevator when I felt it: a sudden,…

Adults and children with severe hemophilia A who are receiving Nuwiq (recombinant human coagulation factor VIII) as their first replacement therapy have a similar risk of developing inhibitors as those treated with plasma-derived therapies containing von Willebrand factor, the final results of the NuProtect trial show.

A single dose of Spark Therapeutics’ investigational gene therapy SPK-8011 increased the levels of factor VIII (FVIII) — the missing clotting protein in hemophilia A — and, in 16 of 18 male patients, those levels were sustained for up to four years, according to results from a Phase 1/2 clinical…

In this video from the Hemophilia Federation of America, Kyle McCullough and Jesse Mayhan share what it’s like living with severe hemophilia. The boys discuss their treatment and what they tell friends when they ask about their condition. They also talk about what they’d like to…

Sarcopenia, or loss of muscle mass and strength, may occur in as many as half of adults with severe hemophilia A who experience recurrent joint bleeds, according to a small study in Turkey. In addition, measuring muscle mass in the thighs with ultrasound, which is a noninvasive imaging test…

The European Medicines Agency (EMA) has agreed to review an application by Laboratoire Francais du Fractionnement et des Biotechnologies, a French biopharmaceutical known as LFB, requesting the approval of eptacog beta for the treatment of bleeding episodes in patients with hemophilia A or B with inhibitors. A…

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…

In the realm of hemophilia, I believe the current classification system oversimplifies a far more intricate reality. Severe, moderate, and mild hemophilia are classifications based on the amount of clotting factor present in a person’s blood. Hemophilia is a genetic disorder that impairs the blood’s ability…

The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended Hemlibra’s (emicizumab) approval be expanded to include people with moderate hemophilia A without inhibitors. “We’re very pleased that the CHMP’s recommendation brings us closer to potentially transforming the day-to-day lives of people in the…

Altuviiio (efanesoctocog alfa) reduced bleeding and was safe for boys and young men with severe hemophilia A, according to a recent study. Researchers also reported an increase in participants’ confidence in everyday activities and no return of neutralizing antibodies that may inhibit treatment, even in patients who are usually…