Pfizer (PFE) Phase III DMD Gene Therapy Study Misses Goal

Pfizer (PFE) announced that a phase III study on its mini-dystrophin gene therapy fordadistrogene movaparvovec for treating Duchenne muscular dystrophy (“DMD”) failed to meet the primary endpoint as well as key secondary endpoints.The global phase III study, CIFFREO is investigating fordadistrogene movaparvovec in patients 4-8 years of age with DMD. A progressive and degenerative disorder, DMD leads to weakness and wasting away of the body’s muscles due to disruption of dystrophin production. The disease us ...