uniQure plans to advance its investigational gene therapy AMT-061 into a pivotal study in 2018 for patients with severe and moderately severe hemophilia B. Gene therapies are being develop to treat diseases caused by genetic mutations. It involves replacing the faulty gene in patients with a healthy gene.
More therapies are now available for the 30 million or so people with rare diseases in the U.S. than ever before, and millions of dollars are being invested in clinical studies that will test new ways of evaluating — and advancing — potential treatments, including the use of natural history…
Researchers have found that the high price of recombinant clotting factors for patients with hemophilia B is the main cause of the economic burden of the disease in the United States. However, despite the cost, prophylaxis treatment can provide clinical benefits to patients that may reduce other costs not…
As Congress begins debate this week to overhaul the U.S. tax code, lawmakers should leave the Orphan Drug Act (ODA) — and the tax incentives it offers pharmaceutical companies to develop therapies for rare diseases — off the table. That’s the message being pushed by the National Organization for…
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 …
Knowledge about the clinical benefits of treatments and their importance in the outcome of patients, as well as more stable therapies, will likely improve treatment adherence among hemophilia patients in the U.S., according to new research. The study, “Treatment adherence in hemophilia,” was conducted by Dr. Courtney Thornburg, medical…
Look out, Phoenix, there’s a whole lot of zombies heading your way. The Arizona Hemophilia Association (AHA) is teaming up once again with Lerner and Rowe’s Phoenix personal injury law offices and the radio station 98KUPD to hold Zombie Walk 9 in downtown Phoenix on Oct. 28. The fundraising event…
U.S. hemophiliacs who use patient-reported outcome (PRO) instruments can provide reliable information on their disease burden, finds a new study that appeared in the journal Patient Preference and Adherence. In the study, “Reliability of patient-reported outcome instruments in US adults with hemophilia: the Pain, Functional Impairment and Quality…
The spleen’s marginal zone (MZ) B-cells respond to blood coagulation factor VIII (FVIII) and show potential to become the target of future therapies to reduce FVIII inhibitors in hemophilia A, according to new research in mice. The study, “Marginal zone B cells are critical to factor VIII inhibitor…
Three U.S. bleeding disorder patient organizations recently received funding support for their patients’ rights advocacy campaigns. The Milwaukee, Wisconsin, headquartered Great Lakes Hemophilia Foundation (GLHF), the New York City Hemophilia Chapter (NYCHC), and the Columbus, Ohio-based Ohio Bleeding Disorders Council (OBDC) have been…
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