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Tag Archives: rare disease therapies

The economics of rare disease therapies

I came of age scientifically at the beginning of the cloning era. As various genes associated with human genetic disorders—sickle cell disease, cystic fibrosis, muscular dystrophy, Huntington’s—were cloned, the papers reporting these successes always ended with some statement that now the door was open to therapy. These prophecies proved to be wildly optimistic. Now, with the advent of CRISPR gene editing, it is becoming possible to realize the...

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