Dr. Timothy Yu walks us through his experience running the first "N of 1" drug trial for a young patient with a devastating genetic mutation.
Every so often, someone comes up with a new idea that is truly visionary. The first time you see it, you may not immediately appreciate how transformational it might become.
As an attending physician in Boston Children's Hospital's Division of Genetics and Genomics, Tim was caring for a young patient with a devastating genetic mutation. He suspected her condition could be treated with an adaptation of an existing class of therapies, and he also knew that she was likely the only person in the world who would benefit from that drug. Instead of accepting the conventional wisdom that N of 1 means you don't have a valid experiment, Tim asked himself a bold question: "Why not try to give her the therapy?"
Tim went to the FDA, created a toxicology package, got it approved, manufactured it, and created a lifetime supply for his patient.
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