WHY SPINRAZA/HOW SPINRAZA WORKS
SPINRAZA® (nusinersen) is delivered directly to the CSF at the site of motor neuron loss
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.
It is delivered to the cerebrospinal fluid (CSF), the area surrounding the spinal cord, allowing it to be distributed to the target tissues. While you continue treatment, SPINRAZA helps your body increase the production of SMN protein.
SPINRAZA targets the underlying cause of muscle weakness in SMA by increasing spinal motor neuron (SMN) protein levels
People with SMA can't make enough SMN protein because they have a mutated or missing survival motor neuron 1 (SMN1)
gene. The gene they do have, SMN2, does not produce enough of the SMN protein that is needed for motor neurons to survive.
