Eloctate (efmoroctocog alfa) for hemophilia - Hemophilia News …
Dec 4, 2023 · Eloctate (efmoroctocog alfa) is an extended half-life therapy that’s approved to prevent and treat bleeds, including those occurring during surgery, in adults and children with hemophilia A. Its design allows it to last for a longer time in the body, thus requiring fewer doses. The therapy, which is administered via an intravenous or into-the ...
FEIBA (factor eight inhibitor bypassing activity) for hemophilia
Nov 9, 2023 · FEIBA (factor eight inhibitor bypassing activity), also known as activated prothrombin complex concentrate and anti-inhibitor coagulant complex, is a bypassing agent that’s approved to prevent or control bleeding episodes in people with hemophilia A and hemophilia B. It can be prescribed for patients with inhibitors, or neutralizing ...
Real-world results with hemophilia B gene therapy
Aug 2, 2024 · HEMGENIX ®, etranacogene dezaparvovec-drlb, is a one-time gene therapy for the treatment of adults with hemophilia B who: Currently use Factor IX prophylaxis therapy, or. Have current or historical life-threatening bleeding, or. Have repeated, serious spontaneous bleeding episodes. HEMGENIX is administered as a single intravenous infusion and ...
Review study shows prevalence, impact of hemophilia in Brazil
by Patricia Inácio, PhD May 31, 2024. In Brazil, hemophilia markedly impairs patients’ quality of life and ability to work, with up to 82.6% of people with the disorder being unemployed, a review study finds. Barriers to treatment include long distances to centers, lack of coordination of specialized and emergency care, and restricted access ...
Hemophilia symptoms: Early signs and complications - Hemophilia …
Oct 18, 2023 · Hemophilia is a rare disorder in which the blood doesn’t clot properly, resulting in the disease’s hallmark symptoms of bleeds — excessive bleeding that extends over a prolonged period of time — that in turn leads to other complications. Bleeds can occur outside the body (external bleeding) or within the body (internal bleeding). In …
New gene-editing strategy may be safer, more efficient
Sep 3, 2024 · A team of U.S. researchers has developed a new gene-editing strategy that could make the approach safer and more efficient for treating certain diseases, such as hemophilia. In preclinical studies, the gene-editing technique was shown to enable fast and efficient delivery of healthy genetic material to cells, while bypassing some of the major ...
FDA Approves Hemgenix, 1st Gene Therapy for Hemophilia B
Nov 23, 2022 · by Margarida Maia, PhD November 23, 2022. The first gene therapy for adults with hemophilia B — branded as Hemgenix (etranacogene dezaparvovec) — has been approved by the U.S. Food and Drug Administration (FDA). Meant to be a one-time treatment, Hemgenix is expected to keep bleeding in check in the long term, while also allowing patients to ...
First hemophilia A patient dosed in trial of BBM-H803 in China
Jan 10, 2024 · The Phase 1/2/3 clinical trial (CTR20233400) is testing how safe and well tolerated BBM-H803 is as a single into-the-vein (intravenous) infusion for adults with severe hemophilia A. The company also plans to test how well it works over 52 weeks, or a year. “Today is a special day for both Belief BioMed and hemophilia A patients, as the first patient dosing has been successfully completed in ...
AlphaNine SD (human coagulation factor IX) for hemophilia
Mar 21, 2024 · What is AlphaNine SD for hemophilia? AlphaNine SD (human coagulation factor IX) is a highly purified, plasma-derived clotting factor IX (FIX) concentrate that’s approved to prevent and control bleeding in people with hemophilia B. The therapy, which is given via an infusion into the vein, or intravenously, was originally developed by Alpha ...
Fighting for proper care as a woman affected by bleeding disorders
Sep 13, 2024 · As a parent of three daughters, Ashley became concerned when one began displaying troubling symptoms, including excessive bleeding after dental extractions. “As a toddler, she had a giant lump on her head,” Ashley recalled. “It was big, purple, and didn’t go away for months.”. Despite these alarming signs, it wasn’t Ashley’s local ...