First results from the Phase 3 HAVEN 2 study evaluating the effectiveness and safety of emicizumab in children younger than 12 with hemophilia A look promising. Roche announced that preventive treatment with emicizumab caused a reduction of the number of bleeds over time, with no major adverse events reported so…
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When given as a prophylactic, or preventive therapy, or on an on-demand basis, FEIBA (factor eight inhibitor bypassing activity) was deemed safe and effective at lessening the number of bleeds in people with severe hemophilia and high levels of inhibitors. That’s according to four-year data from a real-world…
Last updated Nov. 21, 2023, by Margarida Maia, PhD ✅ Fact-checked by Joana Carvalho, PhD What is Obizur for hemophilia? Obizur (susoctocog alfa) is a recombinant, or man-made, antihemophilic factor therapy approved for the on-demand treatment of bleeding episodes in adults with acquired…
The levels of sclerostin, a protein that regulates bone formation, may be linked to disease severity and reflect bone alterations in children with hemophilia A, a study suggests. The study, “Serum Sclerostin Level and Bone Mineral Density in Pediatric Hemophilic Arthropathy,” was published in The Indian…
Advocacy doesn’t always mean protests or politics; sometimes it begins with a parent learning everything they can to protect their child. One mother shares how speaking up for her sons with hemophilia helped teach them to one day speak for themselves.
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Can Hemophilia Be a Disability?
Social media. You either love it or hate it. I must confess that I am guilty of wasting time on Facebook and Twitter, but I have used these outlets as a means of support on more than a few occasions. In the bleeding disorder community, several Facebook groups…
A gene therapy from uniQure in early testing, called AMT-180, has the the potential to treat all hemophilia A patients, including those with inhibitors, according to the company. Hemophilia A is caused by missing or defective factor VIII (FVIII), a clotting protein. About 30% of patients with severe hemophilia A develop inhibitors, or…
The U.S. Food and Drug Administration (FDA) approved Sevenfact (coagulation factor VIIa [recombinant]-jncw) for the treatment and control of spontaneous bleeding episodes in people, ages 12 and older, with hemophilia A or B with inhibitors. Hemophilia, a genetic disorder, affects the body’s ability to make blood…
The U.S. Food and Drug Administration (FDA) recently granted orphan drug status to SHP654 (BAX 888), an investigational factor VIII (FVIII) gene therapy candidate being developed by Shire for the treatment of hemophilia A. Gene therapy allows the delivery of a functional copy of the defective gene.
Hemophilia C is a rare bleeding disorder that affects the blood’s ability to clot.