About

Duchenne muscular dystrophy is a severe muscle-wasting disease that typically begins in early childhood, rapidly progresses to loss of walking ability, and weakens muscles in the legs, pelvis, and arms. Z-Rostudirsen is designed to deliver a therapeutic agent that prompts muscle cells to skip over a specific flawed section of the dystrophin gene, called exon 51. This skipping may allow the production of a more functional dystrophin protein, which could potentially slow the progression of muscle degeneration in these patients.