In the hustle and bustle of daily life, we often overlook the extraordinary within the ordinary. My husband, Jared, has hemophilia, and we’ve found that living with the bleeding disorder has a way of sprinkling our lives with one-of-a-kind situations that might seem mundane to us but are…
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Electrically stimulating the vagus nerve — the main set of nerve fibers that control involuntary bodily functions — significantly reduced blood loss and sped up clot formation after injury in a mouse model of hemophilia A, according to a recent study. Controlling bleeding by stimulating the vagus nerve was…
An experimental gene therapy using modified blood stem cells helped five adults with severe hemophilia A attain therapeutic levels of clotting factor VIII (FVIII) to prevent and control bleeding, according to a small first-in-human study. Over the course of the Phase 1 clinical study (NCT05265767), none of the…
Hemophilia is a disorder characterized by excessive bleeding. Aminocaproic acid, also known as 6-aminocaproic acid or epsilon-aminocaproic acid, is an antifibrinolytic medication that can help to stabilize blood clots.
Refixia or Rebinyn (nonacog beta pegol, or N9-GP), an approved replacement therapy for hemophilia B, is effective and has a good safety profile in previously untreated boys younger than 6 with severe to moderately severe disease, an early analysis of a Phase 3 trial indicates. In…
Hemophilia patients are taking heart from a number of innovative treatment approaches that have reached the clinical trials stage. Researchers at the Children’s Hospital of Philadelphia offered a rundown on the advances in an article in the journal Blood titled “Novel approaches to hemophilia therapy: successes and challenges.” They include …
Albumin fusion is a molecular technology that has been used to create replacement therapies for hemophilia with an extended half-life — treatments that are more long-lasting in the body and can therefore be administered less frequently.
Novo Nordisk’s Refixia (nonacog beta pegol, N9-GP) received a positive opinion by the Committee for Medicinal Products for Human Use (CHMP) for treatment of adolescents and adults with hemophilia B. The CHMP, under the European Medicines Agency (EMA), recommends the granting of a marketing authorization in Europe for…
The effectiveness of liver-directed, viral-based gene transfer — the most used experimental gene therapy approach for hemophilia A — is significantly reduced in mouse models of hemophilia A relative to healthy mice, a study shows. This limitation was associated with a pronounced reduction in fenestrations — cell surface…
UniQure‘s investigational gene therapy AMT-060 for severe hemophilia B has received PRIME designation by the European Medicines Agency (EMA). The EMA designation was based on the results of a Phase 1/2 open-label, dose-escalating study of AMT-060 (NCT02396342). The study included 10 patients, each receiving…