Sarepta gave the one-time drug a price tag of $3.2 million, making it the second most-expensive medicine in the world. DMD affects one in 3,500 to 5,000 newborns, mostly boys because of the way it’s inherited, according to Johns Hopkins Medicine. The primary goal of the confirmatory trial was improvement on a measure known as the North Star Ambulatory Assessment, a scale of how well kids can stand, walk and perform other movements. The difference wasn’t statistically significant, meaning technically the study failed to meet its main goal. The company said no new safety issues emerged in the trial, which it called EMBARK.

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Persons: Doug Ingram, ” Ingram, Sarepta, “, Dr, Sanjay Gupta, Ingram, ” Organizations: CNN, US Food and Drug Administration, Sarepta Therapeutics, FDA, Johns Hopkins Medicine, North Star, CNN Health