Pfizer’s program, as well as a rival treatment from Sarepta Therapeutics, are among the most closely watched in all of gene therapy research.
Both are in the final stages of testing and have shown they can help Duchenne patients produce a shortened version of the shock-absorbing protein, dystrophin, that they lack. Those findings have raised hope that each could change the course of a progressive and deadly disease that typically leaves patients in a wheelchair by their teenage years and kills them at a young age.
