Comment Re:Exon Skipping? (Score 2) 264
According to NPR, Kalydeco, "works by helping to fix one defect in the protein that causes the disease." Unfortunately the way the drug works is also very specific, and won't work for all sufferers NPR also reports that it will "only work for about 1,200 patients in the U.S.". Now, being that a cursory Internet search says that there are about 30,000 sufferers in the USA, it's pretty clear that Kalydeco is just a step in the right direction, at least from a medical and research perspective.