Onasemnogene abeparvovec

Onasemnogene abeparvovec is a gene therapy treatment designed for infants with spinal muscular atrophy (SMA), a genetic disorder that weakens muscles and affects movement. It works by delivering a healthy copy of the faulty gene responsible for SMA directly into the child's cells using a modified virus. This helps the body produce the missing or defective protein needed for muscle strength and function. Administered as a one-time intravenous infusion, the therapy aims to improve survival and motor skills, offering a potential to change the disease's progression if given early in the disease course.