4min chapter

Freakonomics, M.D. cover image

72. What’s Stopping Us From Curing Rare Diseases?

Freakonomics, M.D.

CHAPTER

CRISPR Medicines - The First Step in a Four Year Process to Protect Patients From Failed Medicines

For many diseases, that engineered CRISPR on my computer screen can become a vial with that CRISPR that we can pretty quickly test for whether it can repair the defect safely and effectively. Using the medicine is the first step of probably a four year process to protect patients from faulty medicines. Many of these diseases are individually so rare that they do not form a viable commercial proposition under the current system. If you have a new technology that will allow for personalized intervention in people with life-threatening diseases which early treatment really does matter, we should be able to create a regulatory pathway"

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