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Pfizer’s experimental gene therapy to treat Duchenne muscular dystrophy appeared to improve muscle function in a study of nine boys, but also resulted in serious drug reactions in three of those children, the company said Friday.

The data are being presented at a virtual session of the American Society of Gene & Cell Therapy.


Duchenne, which affects 12,000 children in the U.S., almost all of them boys, is a genetic disease in which muscles degenerate due to lack of a key protein called dystrophin, eventually leading to death. Several companies have been developing gene therapies that work by using viruses to sneak a truncated, but still functional, protein into cells.

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