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…
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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…
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.
Supporters of World Hemophilia Day, on April 17, are hoping to raise awareness about the bleeding disorder with events planned globally and on social media, and to advocate for sustainable and equitable access to care and treatment. The annual event, which is organized by the World Federation of Hemophilia…
Severe hemophilia A patients using Esperoct (turoctocog alfa pegol) as a preventive treatment do well, with fewer bleeds or breakthrough bleeds, lower costs, and better life quality compared with other extended half-life (EHL) therapies, a modeling analysis suggested. The…
My eldest son, Julian, is 22. He is studying music at college. It’s his passion, and as I have a music education degree and my husband has a music degree, his course choice did not come as a surprise. With our recent move, Julian changed schools, and his…
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.
The Phase 1/2 trial looking at the Factor VIII protein SHP656 as a long-acting therapeutic to treat hemophilia A did not meet a pre-defined once-weekly dosing criterion, according to Xenetic Biosciences and Shire, which are jointly developing the drug. The study demonstrated SHP656’s efficacy and pharmacokinetic data (the drug’s behavior once…
I have von Willebrand disease (VWD) and hemophilia B, a less common form of hemophilia that arises when a blood-clotting protein called factor IX is missing or deficient. Hemophilia is often thought of as a condition that only affects men, but women can and do have it, too.
Jivi was safe and effective at preventing and treating bleeds for more than five years in boys under age 12 with severe hemophilia A, according to long-term data from a Phase 3 trial’s extension study. Data analysis also showed that most of…