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" ... From the start, the Sarepta story has been a strange case. It was incredibly expensive to make–in small batches probably costing $100,000 per patient, analysts say–and the company was strapped for cash. So it did a small study of 14 patients. Two of those patients were not evaluable, but in the rest something surprising seemed to happen: those that got Exondys 51 from the start of the study seemed to do much better than those who had a delayed start on the drug. More enticingly, the boys (because the genetic defect that causes Duchenne is on the X chromosome, it is a disease of boys) seemed to be showing marked increases in dystrophin, the protein that is missing in the disease. ... "