BioMarin Pharmaceutical has completed enrollment in GENEr8-3, a Phase 3b study evaluating its investigational gene therapy Roctavian (valoctocogene roxaparvovec) in combination with corticosteroids in men with severe hemophilia A. Top-line data from the 52-week (one year) study are expected to be released in early 2023, according to a…
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Alhemo (concizumab-mtci) is an antibody-based therapy that is approved to prevent or reduce the frequency of bleeds in patients with hemophilia A or B, with inhibitors. It is given as daily under-the-skin, or subcutaneous, injections that can be self-administered.
I just returned from the Hemophilia Federation of America (HFA) Symposium in New Orleans, and like many gatherings in the bleeding disorders community, it left me inspired, reflective, and deeply grateful. This was my first HFA Symposium, and I’m truly grateful to the organization for including me. There’s something…
Preventive treatment with Hemlibra (emicizumab) was not considered cost-effective at its current price compared with standard replacement therapies in adults with mild or moderate hemophilia A without inhibitors, according to a modeling study. The cost of Hemlibra would need to drop by at least 35% to become cost-effective…
“Whiskey-Bravo-six-Zulu-Yankee-Yankee looking for a radio check,” my dad called out into his faithful Kenwood handheld 2-meter radio. “W-B-6-Zed-Y-Y, I hear you loud and clear,” came an answer from radio land. This was my dad’s call sign — the constant background noise of my youth. Ask any childhood friend who spent…
Researchers at Emory University School of Medicine have developed a small-scale model system to study bleeding and clotting of wounds. The miniature model also accurately reproduces the alterations observed in hemophilia A patients. Their work, “A microengineered vascularized bleeding model that integrates the principal components of hemostasis”, was published in…
What was my reaction the first time I encountered a comprehensive hemophilia treatment center (HTC)? Relief! I couldn’t believe the resources that were available to those fortunate enough to be treated at one. With quality care, emotional and employment counseling, physical therapy, dental health, and school assistance, the HTCs seemed…
Nuwiq (simoctocog alfa) is an approved therapy that works to prevent and control bleeding episodes, including those occurring during surgery, in adults and children with hemophilia A.
A Phase 3 clinical trial of treatment candidate AMT-061 for severe and moderately severe hemophilia B has treated its first patient. The open-label HOPE-B study (NCT03569891) is testing the safety and effectiveness of AMT-061 in adult men with hemophilia B. Patient recruitment is continuing for an estimated total of 56…
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Baudax Bio’s investigational regulatory T-cell therapy TI-168 for the treatment of hemophilia A with inhibitors. Orphan drug status is given to therapies intended to treat rare conditions, defined as those affecting less than 200,000 people in…