About

Duchenne muscular dystrophy is a severe muscle-wasting disease that begins in early childhood, rapidly progresses to loss of walking ability, and can affect the heart and lungs. Z-Rostudirsen is an investigational drug designed to skip over a faulty section of the dystrophin gene, called exon 51, to help muscle cells produce a more functional version of the crucial dystrophin protein. This approach may potentially slow the relentless muscle degeneration that defines the disease.