How many patients could benefit from custom genetic therapies? Timothy Yu narrows in on an answer
Timothy Yu sent shockwaves through the genetic therapy world four years ago after revealing that he had developed — in under a year — a treatment tailored to a young girl’s unique genetic mutation.
That drug, dubbed Milasen in honor of the girl Mila, raised the bar on how truly personalized a medicine could be, and ignited efforts to develop the so-called n-of-1 therapies for more patients. Yet there was one question that kept nagging at Yu: How many others like Mila were out there?
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