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A single dose of Spark Therapeutics’ investigational gene therapy SPK-8011 increased the levels of factor VIII (FVIII) — the missing clotting protein in hemophilia A — and, in 16 of 18 male patients, those levels were sustained for up to four years, according to results from a Phase 1/2 clinical…

Idelvion (albutrepenonacog alfa) is an approved factor IX (FIX) replacement therapy for the prevention and treatment of bleeds, including those occurring during surgery, in adults and children with hemophilia B.

I live in the small town of Punta Gorda, Florida. Though I have hemophilia B and von Willebrand disease, I don’t know anyone here or in the surrounding Charlotte County with a bleeding disorder. While living in such a community can present challenges when dealing with…

SPK-8011, also known as dirloctocogene samoparvovec, is an experimental gene therapy for hemophilia A. Given as a one-time intravenous or into-the-vein, infusion, it aims to reduce the risk of bleeds in patients.

Despite high rates of bone health issues in hemophilia and von Willebrand disease (VWD), the routine use of DEXA scans remains low across hemophilia treatment centers (HTCs) in the U.S., a study reports. DEXA, or dual-energy X-ray absorptiometry, is a type of imaging test that uses low-dose X-rays to…

Sevenfact (eptacog beta) is a bypassing agent that’s approved to treat bleeding episodes in people with hemophilia A or hemophilia B who developed inhibitors, or neutralizing antibodies, against clotting factor VIII (FVIII) or factor IX (FIX).