nusinersen (Spinraza)

Nusinersen (Spinraza) is a medication designed to treat spinal muscular atrophy (SMA), a genetic disorder that causes weak muscles and movement problems due to loss of nerve cells controlling muscles. It works by increasing the production of a critical protein needed for motor neuron survival, helping to improve muscle strength and function. Administered through injections into the spinal fluid, Spinraza can slow disease progression and improve quality of life in affected individuals. It is the first approved treatment targeting the underlying cause of SMA, offering new hope for patients with this condition.