What Should You Know Waiting for Gene Therapy?

For many people suffering from rare diseases, gene therapy offers the hope for relief and perhaps a cure for their particular condition.

But while many studies are underway, to date, few drugs have made it through the pharma pipeline and onto the market. That doesn’t stop the pharmaceutical industry from chomping at the bit–just the word “therapy” is marketing gold.

Scientists are seeing an influx of financial support, and investors are getting ready to count their profits. But are they putting the horse before the cart? What about the patients?

If only gene therapy was really this straight-forward. Source: Blogspot

For most laymen, gene therapy is of interest, but for different reasons: Can it be used to grow giant cows and increase beef production? Is Dr. Frankenstein in his lab plotting the introduction of the world’s first super-human?

Researchers recognize the fact that, for many people, gene therapy is considered a novelty at this point in time. To successfully treat patients with this new science, there is going to have to be a campaign of awareness and education, with information being disseminated not only to the payers, but also to the public in order to move forward.

For better or worse, people should know super-powered cows aren’t really what the industry’s going for. Source: AnimalsLook

Patients are more empowered than ever before, and there is greater awareness about clinical trials. They deserve to know that, for them, gene therapy could be life-saving–it’s not just about cows or looking 20 years younger.

So, how are pharma and private companies, such as those who provide DNA tests for family historians or for determining a person’s genetic risk of cancer, changing the landscape? It’s hard to say because the science of gene therapy evolves on a daily basis, but we are seeing more of a ramp-up among Big Pharma, payers, and private industry.

Meanwhile, the people patiently wait for gene therapy to hit the market. Source: Healthin30

There isn’t any schedule for the release of gene therapy drugs, but of paramount importance to the scientific community is the question: Are these products safe? Unfortunately for the patients who await new treatments, the answer is: Only time will tell.


Erica Zahn

Erica Zahn

Erica Zahn is passionate about raising awareness of rare diseases and disorders and helping people connect with the resources that may ease their journey. Erica has been a caregiver, and is a patient, herself, so she completely relates to the rare disease community--on a deeply personal level.

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