Biotechnology company Regenxbio announced Wednesday that preliminary findings from their clinical trial demonstrate ongoing enhancement of muscle function in patients receiving their investigational gene therapy for Duchenne muscular dystrophy.
The company reported that the early-to-mid stage research revealed their experimental treatment maintained a favorable safety record while continuing to show positive effects on muscle performance in study participants diagnosed with the genetic disorder.
Duchenne muscular dystrophy is a progressive muscle-wasting disease that primarily affects boys and typically leads to significant disability and shortened lifespan.
