An experimental Pfizer (PFE) gene therapy appears to help children with Duchenne muscular dystrophy, a lethal muscle-wasting disease that affects as many as 12,000 American boys, according to a small but closely watched study that will be presented Friday.
Two of the six boys who had been treated with the gene therapy for a year or more performed better on a test of muscle function, but its side effects were severe enough that two other boys were hospitalized.
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