A gene therapy that can treat patients with severe hemophilia A is likely to be more cost-effective over the long term than reliance on prophylactic (preventive) therapy using factor VII (FVIII), a model-based analysis of the two treatment approaches reports. The study, “Gene therapy in hemophilia A: a cost-effectiveness…
Precision BioLogic revealed promising data on a new kit for the measurement of FVIII inhibitors (factor VIII) in patients with hemophilia A. The results were presented at the International Society on Thrombosis and Haemostasis’ Scientific and Standardization Committee (SSC) meeting July 18-21 in Dublin, Ireland. Researchers presented their…
Hemophilia patients may now benefit from financial assistance for out-of-pocket expenses, health insurance premiums, and incidental medical costs following the launch of a new program from The Assistance Fund (TAF). Founded in 2009, TAF is an independent nonprofit aimed at helping eligible patients and their families cover costs not…
BAY 94‐9027, an investigational factor VIII replacement therapy being developed by Bayer for severe hemophilia A, is able to stay longer in patients’ blood than other standard therapies, according to a study analyzing data from three…
Pfizer has partnered with Spark Therapeutics to launch a Phase 3 program to evaluate the safety and effectiveness of its factor IX replacement gene therapy for treating hemophilia B. Replacement of factor IX (FIX), the clotting protein lacking in hemophilia B, will be done via the fidanacogene elaparvovec gene…
XaTek announced that it has raised $9.1 million to advance the development of ClotChip — a portable test designed to rapidly analyze, using a single drop of blood, the ability of a person’s blood to clot. The capital was raised in a series A financing round, which marks a company’s first significant…
BioMarin Pharmaceutical Inc. is partnering with digital content agency Believe Limited to produce a three-day musical workshop for young members of the bleeding disorders community. The “Breaking Through!” musical theater intensive, the first program of its kind, will be held Nov. 9-12 in New York City, and aims at teaching…
A panel of multidisciplinary experts have developed recommendations for applying best practice guidelines in the planning and managing of surgery in hemophilia patients. The guidelines were outlined in the consensus review, “Recommendations on multidisciplinary management of elective surgery in people with haemophilia,” published in the journal…
Although moderate to severe hemophilia patients are generally protected against the development of cardiovascular disease, cardiovascular-related events can still occur in this patient population, a study reports. These findings suggest that efforts should be made to prevent cardiovascular disease in hemophilia patients who have risk factors associated with the…
Key leaders in hemophilia therapy development, representing large pharmaceutical and biotech firms as well as academia and contract research organizations, will gather at the Hemophilia Drug Development conference in Boston on Aug. 14 through 16. The meeting is designed to be an industry-dedicated networking and discussion forum, and many of…
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