ZOLGENSMA® (onasemnogene abeparvovec-xioi) is a prescription gene therapy used to treat children less than 2 years old with spinal muscular atrophy (SMA). ZOLGENSMA is given as a one-time infusion into the vein.
ZOLGENSMA is designed to target the genetic root cause of SMA by replacing the function of the missing or nonworking survival motor neuron 1 (SMN1) gene.
The new gene tells motor neuron cells to produce more survival motor neuron (SMN) protein. Motor neuron cells need SMN protein to survive and support muscle functions.