Onasemnogene abeparvovec is a gene therapy designed to treat spinal muscular atrophy (SMA), a genetic disorder that affects motor neurons and leads to muscle weakness and atrophy. This therapy works by delivering a copy of the SMN1 gene, which is missing or mutated in individuals with SMA, to help produce the necessary protein for motor neuron health. Administered as a one-time intravenous infusion, onasemnogene abeparvovec aims to improve motor function and increase survival rates in patients, particularly in those with the most severe form of SMA. It represents a significant advancement in the treatment of this condition, offering hope for affected individuals and their families.