Still reeling from the deaths of two patients taking its Duchenne muscular dystrophy gene therapy Elevidys and the black box warning that resulted, Sarepta said late yesterday that a patient in an early-stage trial of one of its limb-girdle
muscular dystrophy candidates had also died. Gene therapy promises huge rewards, but development is not without risk, and Sarepta has paused development of this candidate, among others. |