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Game Changer: Gene Therapy Effective in Hemophilia B

November 20, 2014; Posted by: WeBleed staff

Research in gene therapy succeeding in those with hemophilia B.


Good news for the those who have hemophilia B. According to a new report outlined in the New England Journal of Medicine, 10 patients with severe hemophilia B have remained cured of the inherited bleeding disorder for three years in large part to gene therapy. In order to engineer the virus to treat the 10 patients required six months of work and the main problem is the need to have better methods to produce the virus to treat hemophilia B.

“I believe that, scientifically, this is ready for prime time,” the chief author, Dr. Andrew Davidoff of St. Jude Children’s Research Hospital in Memphis, Tennessee said in an article by Fox News. “Hopefully (the new results) will be a catalyst for us and others to find a more effective way to produce (the virus),” Davidoff said. “In fact, we’re in discussions with other companies in terms of investing in our research for improving production.”

So far, the therapy has had no serious side effects and is designed to eliminate the need to give patients injections of factor IX every two or three days at a cost of about $250,000 per year.

“I think it’s going to have a big impact. The study showed both safety and efficacy and the side effects were minimal,” said Dr. Timothy Nichols, who heads the Francis Owen Blood Research Laboratory at the University of North Carolina. “This is a single shot of medicine given to patients who are treating themselves two or three times a week. Suddenly, they don’t have to take the medicine anymore.”

Great news for the bleeding disorders community. Click here to read the full article.

Photo Credit – Fox News – your source for bleeding news!

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