Hemophilia B patients living in urban mainland China have longer hospitalizations and significantly higher medical costs — including more expensive fees for coagulation factors concentrates — compared with patients with hemophilia A, a retrospective study shows. In fact, people with hemophilia B had inpatient medical costs more than…
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Roctavian (valoctocogene roxaparvovec-rvox) is an approved one-time gene therapy that’s intended to reduce the risk of bleeds in certain adults with severe hemophilia A.
My Facebook friends fall into a few categories: biological family, colleagues and friends from my music and teaching days, high school and college friends and teachers, and my “bleedin’ folk” family. Raising two sons with severe hemophilia is a blessing because of the people in my life (I work…
Hemgenix (etranacogene dezaparvovec), the first approved hemophilia B gene therapy, has been successfully administered to two patients in France, marking the first time the treatment has been given in a real-world setting in Europe. The gene therapy, originally developed by uniQure and marketed by CSL Behring, was…
The U.S. Food and Drug Administration (FDA) has granted fast track designation to SerpinPC, Centessa Pharmaceuticals’ investigational treatment for people with hemophilia B. Fast track status expedites the review of therapy candidates that show a potential to fill an unmet medical need for serious conditions. It also allows more…
I recently visited the small town of Arcadia, Florida, which is not far from my home. Arcadia holds an important place in history for the topic of hemophilia and AIDS. The story of what happened there is a lesson about how ignorance can fuel hatred and knowledge…
Bone mineral density, or the amount of mineral in bone tissue, is decreased in 63.1% of people with hemophilia, according to a study from Germany. Study findings also indicated the degree of loss depended on hemophilia severity. Patients with severe hemophilia had lower bone mineral density in the left…
GS1191, a gene therapy in the pipeline of Gritgen Therapeutics, increased factor VIII (FVIII) activity in the blood of people with hemophilia A taking part in a small clinical trial in China, resulting in fewer bleeding episodes. The fully enrolled investigator-initiated trial (ChiCTR2300073179) is testing the safety and…
Concizumab has been approved as a preventive treatment in Canada for hemophilia B patients ages 12 and older who are positive for factor IX inhibitors and need a routine prophylactic to prevent bleeds or reduce their frequency. This is the first approval for the daily under-the-skin injectable therapy, which…
In an article titled “Recognizing the need for personalization of hemophilia patient-reported outcomes in the prophylaxis era” published in the scientific journal Haemophilia, researchers reviewed two new tools that are used to measure patient reported outcome measures.