Why This New Hemophilia Approach is Revolutionary

Gene therapy is the hot new thing in medicine these days–especially with hemophilia.

The good new is, “Yay! There are hot new things in medicine exciting researchers!” The bad news is that a lot of the results have been less than encouraging.

But in the hemophilia world, things are looking pretty good when it comes to treatments that involve gene therapy. With a goal of allowing a person who has hemophilia to live as if they didn’t have the disease, researchers are making treatment headway—at least with hemophilia B.

For those who don’t know, hemophilia is categorized as either type A or B based on which blood clotting protein (known as “factor”) is missing or defective.

In type A, which is four times more common, the issue is with blood-coagulation factor VII (seven, in Roman numerals). It used to be called proconvertin.

For people with type B, the issue lies with factor IX (9).

Fun fact: Factor IX is also called Christmas factor because it was discovered after a patient named Stephen Christmas.

But back to gene therapy breakthroughs…

After about 20 years of false hopes and starts with treatments involving manipulating genes, a study involving gene transfer in 2014 showed positive long-term results with hemophilia B. Now, doctors at the Children’s Hospital in Philadelphia are feeling pumped that a treatment paradigm-shift can be achieved in our lifetimes.

This is information that you really want to pay attention to, so go read the original article here.



EmpatheticBadass is a young-at-heart writer from Ohio (Go, Bobcats & The Marching 110!)) who is passionate about being a voice for the patient perspective.

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