In addition to its clinical challenges, hemophilia is characterized by a major economic burden that affects patients, care givers, the healthcare system and society. In a recent report, Sheh-Li Chen, PharmD, from the University of North Carolina Medical Center, identifies the most significant areas of healthcare hemophilia management, highlighting the importance of the…
The European Medicines Agency (EMA) is considering lowering the minimum number of patients required in clinical trials of potential hemophilia treatments because of “dramatic” increases in hemophilia research and concerns about the availability of patients for rare diseases like hemophilia A. “EMA began the process after becoming concerned that the minimum…
Genentech recently presented positive Phase 1/2 extension results for its investigational hemophilia A medicine, emicizumab, at the 2016Â World Federation of Hemophilia (WFH) World Congress in Orlando, Florida. Emicizumab is being developed by Genentech (a Roche company) and Chugai Pharmaceutical. The latest data analysis continued to show a promising profile…
Researchers analyzed the patterns of bruising in children younger than six years old to understand the extent to which their inherited bleeding disorders, such as hemophilia, affected the number, size, and location of these bruises — and found that bruising in the youngest of these children may be an early…
Researchers have created a model for how factor VIII replacement concentrates behave in the body of hemophilia A patients undergoing surgery, which could allow doctors to calculate more accurate and individualized doses for factor replacement treatment. The study “A Population Pharmacokinetic Model For Perioperative Dosing Of Factor VIII In Hemophilia A…
Shire recently announced that the U.S. Food and Drug Administration (FDA) has approved its Baxject III reconstitution system for Adynovate, an approved treatment for Hemophilia A. This system will allow patients and caregivers to prepare the therapy with fewer steps, minimizing the chances for possible errors. Adynovate is a recombinant Factor VIII (rFVIII) treatment with…
uniQure N.V. has updated the clinical data from its ongoing clinical trial of a gene therapy for the treatment of patients with hemophilia B. The results indicate sustained improvement by all patients at a low-dose, with durable levels of Factor IX (FIX) gene activity for several weeks after treatment. The…
BioMarin Pharmaceuticals presented positive interim data from an open-label Phase 1/2 clinical trial of BMN 270, an investigational gene therapy for severe hemophilia A. The promising results, presented at the XXXII International Congress of the World Federation of Hemophilia (WFH), showed that patients had improved and sustained clotting function. BMN 270 is designed to alter…
Bayer announced that six hemophilia clinicians and researchers in the United States were among 16 grant recipients under the company’s Hemophilia Awards Program for 2016. The announcement was part of the World Federation of Hemophilia 2016 World Congress now taking place in Orlando, Florida. Grant amounts for the six were not given,…
New Phase 3 trial results of CSL Behring’s hemophilia B therapy candidate Idelvion show promise in terms of reduction of bleeds, prolonged usage, and effectiveness at low doses. The poster detailing the data, “Long-term safety and efficacy of recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) in previously treated patients…
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