SPINRAZA (nusinersen)

SPINRAZA® (nusinersen) is a prescription medicine used to treat spinal muscular atrophy (SMA) in pediatric and adult patients. SMA is known as a neuromuscular disease because it originates in the central nervous system (CNS) and affects the motor neurons that power the muscles in the body.

SPINRAZA works by specifically targeting an underlying cause of muscle weakness in SMA.

People with SMA can’t make enough SMN protein, the protein their motor neurons need to function, because they have a mutated or deleted survival motor neuron 1 (SMN1) gene. The

gene they do have, SMN2, does not produce enough protein for all of the body’s muscles. That’s where SPINRAZA can help.