Pfizer’s Gene Therapy Holds Promise in Treatment of DMD, Early Data Show

PF-06939926, Pfizer‘s experimental gene therapy for the treatment of patients with Duchenne muscular dystrophy (DMD), shows promising safety and efficacy results in a small group of boys with DMD, a Phase 1b trial shows. The company presented the preliminary findings at the 25th Annual Parent Project…

Acceleron’s ACE-083 Therapy Candidate for FSHD Earns FDA’s Fast Track Designation

The U.S. Food and Drug Administration (FDA) recently granted fast track designation to Acceleron Pharma’s ACE-083 for the treatment of facioscapulohumeral muscular dystrophy (FSHD). ACE-083 is a locally-acting agent that binds to and inhibits proteins in the transforming growth factor (TGF)-beta family, such as myostatin, which reduce…

Acceleron Therapy Increases Facioscapulohumeral Dystrophy Patients’ Muscle Mass, Trial Shows

Acceleron Pharma’s treatment for a muscular dystrophy affecting the face and other muscles increased the muscle mass of those with the disorder, according to a Phase 2 clinical trial. The results covered the first stage of the trial, which is assessing ACE-083’s3 ability to treat  facioscapulohumeral dystrophy, or FSHD. FSHD…