Will Pfizer’s muscular dystrophy gene therapy cross the finish line?
Others have tried and failed — could this one yield a happy ending for patients?
In early 2021, a young boy in Barcelona was the first to receive Pfizer’s investigational gene therapy for Duchenne muscular dystrophy (DMD). This was part of a global Phase 3 human trial to test Pfizer’s lead candidate, PF-06939926, in a cohort of 99 patients with…