Zolgensma (onasemnogene abeparvovec)

Zolgensma (Onasemnogene abeparvovec) is a gene therapy used to treat spinal muscular atrophy (SMA), a genetic disease that affects motor neurons in the spine, leading to muscle weakness and atrophy. It delivers a healthy copy of the SMN1 gene, which is missing or defective in SMA patients. This therapy aims to restore the production of a vital protein needed for motor neuron function. Approved for children, especially those under two years old, Zolgensma provides a potentially transformative treatment, addressing the root cause of the disease rather than just its symptoms.