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Fifteen children born with a rare muscle-wasting disease would probably not be alive today if not for an experimental treatment that tweaked their genes shortly after they were born.

The disease, called spinal muscular atrophy, would likely have killed most of them by age two. Amazingly, many of the children can now speak and sit on their own, and a few are even walking—milestones they never would have reached without the treatment. The company behind the drug, AveXis, is seeing the most dramatic results in children who are treated in the first month or two of life.

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