Roche has discontinued development of emugrobart, an experimental drug for spinal muscular atrophy (SMA), following disappointing clinical trial results. The company informed European patients in a letter that the therapy failed to consistently improve muscle growth and motor function in a key study.
Spinal muscular atrophy is a rare genetic disorder that affects the motor neurons controlling muscle movement, leading to progressive muscle weakness and atrophy. The condition primarily affects infants and children, making effective treatments critically important for patient families and the broader rare disease community.