STAT Plus: Pfizer, with early however hopeful outcomes, leaps into the race for a Duchenne gene remedy

An experimental Pfizer (PFE) gene remedy seems to assist youngsters with Duchenne muscular dystrophy, a deadly muscle-wasting illness that impacts as many as 12,000 American boys, in keeping with a small however intently watched examine that shall be introduced Friday.

Two of the six boys who had been handled with the gene remedy for a yr or extra carried out higher on a take a look at of muscle operate, however its unwanted side effects had been extreme sufficient that two different boys had been hospitalized.

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