Altuviiio (efanesoctocog alfa) for hemophilia
WEBAug 22, 2024 · Previously known as BIVV001, it’s administered via an into-the-vein (intravenous) injection. It was originally developed by Bioverativ, but the company was acquired in 2018 by Sanofi, which continued Altuviiio’s development in collaboration with Sobi.Sanofi has development and commercialization rights to the therapy in the U.S., while Sobi is responsible for developing and marketing it in ...
Adynovate (rurioctocog alfa pegol) for hemophilia
WEBOct 10, 2023 · How does Adynovate work? Hemophilia A is caused by a faulty or missing clotting FVIII, a protein found in the blood. FVIII is needed for blood to form clots and stop bleedings. Without FVIII, people with hemophilia A may experience heavy, longer-than-usual bleeding episodes occurring either spontaneously …
6 Famous People With Hemophilia – Hemophilia News Today
WEBAug 24, 2017 · Hemophilia is a rare genetic bleeding disorder that mostly affects males, although women can be carriers. Hemophilia has affected people from all walks of life including royalty, sports legends, actors and advocates. Here are just a few famous names who have either had hemophilia or are currently living with the condition, according to The Missing Factor and bloodfeed.com.
Hemophilia prognosis and life expectancy
WEBHemophilia is classified as mild, moderate, or severe, depending on the level of clotting factor in the blood. About 70% of those with hemophilia have the severe form. The most important life-threatening complications of the disease are bleeding in the skull and hemorrhages in soft tissue around airways or other internal organs.
FDA OKs first-in-human study of F9 gene therapy for hemophilia B
WEBFeb 28, 2024 · The announcement of the planned launch of the Phase 1 clinical trial, in an Intellia financial report, follows the recent approval by the U.S. Food and Drug Administration (FDA) of an investigational new drug (IND) application submitted by the two companies. That IND application requested the regulatory agency’s permission to advance the F9 gene therapy into clinical testing.
Roctavian nearly zeroes out bleeds, prophylaxis need over 7 years
WEBAug 20, 2024 · While most of the 12 patients remained off preventive therapy, two returned to regular prophylaxis with factor replacement therapy or Hemlibra (emicizumab-KXWH) due to spontaneous bleeding episodes and declining clotting protein levels.. These data, presented earlier this year in a scientific poster at a European congress, were detailed in the study, “Valoctocogene roxaparvovec gene therapy ...
Real-world results with hemophilia B gene therapy
WEBAug 2, 2024 · A game changer for patients with hemophilia B. Andrea was in her current role as a nurse practitioner at a Wisconsin hemophilia treatment center when she first heard about HEMGENIX ® (etranacogene dezaparvovec-drlb), and she was impressed.FDA approved in 2022 and studied since 2018, it caught her attention as a promising single-infusion treatment for adult male hemophilia B patients that ...
Hemophilia News Today Home | Hemophilia News Today
WEBOct 2, 2024 · Immunotherapy helps suppress inhibitors in acquired hemophilia A. Immunotherapy is an effective treatment strategy to suppress the production of autoantibodies and induce remission in people with acquired hemophilia A, a small study in China suggests.
New gene-editing strategy may be safer, more efficient
WEBSep 3, 2024 · Process uses engineered enzyme to insert therapeutic genes into DNA. The process employs an engineered enzyme called “super-active integrase” that’s able to precisely and efficiently insert therapeutic genes into …
Fighting to be heard on my journey with bleeding disorders
WEBAug 16, 2024 · But every year, during my annual appointments at the HTC, I voiced concerns that my treatment plan wasn’t working.Despite following the regimen, I was hospitalized several times due to post-procedure bleeding. I also struggled with anemia and my menstrual bleeding was severe and unmanageable.. I felt unheard and so frustrated that I’d go home and cry, wondering if it was all in my head.