Medical Report: Cost, not science may be barrier to treatment for some gene therapies

Gene Therapy
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PHILADELPHIA (KYW Newsradio) — Gene therapy is leading to cures and treatments in numerous medical specializations, but the cost remains a barrier.

According to an article in the New England Journal of Medicine, the barrier to treating certain conditions may not be scientific development but cost of treatment — a problem seen with genetic therapy.

A case in point is factor IX (FIX) deficiency, also known as hemophilia B. People with this condition can experience prolonged bleeding, and blood can ooze internally into joints and deep muscles.

A new treatment called etranacogene dezaparvovec has been approved by the FDA as a one-time treatment for adults. One problem: The cost of the one-time treatment is estimated to be $3.5 million.

The article cites numerous examples like this and stresses the importance of making these genetic therapies affordable.

Get more medical reports from Dr. Brian McDonough.

​​KYW Newsradio’s Medical Reports are sponsored by Independence Blue Cross.

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