WHY SPINRAZA/HOW SPINRAZA WORKS
SMA is a disease of the CNS.
SPINRAZA is delivered
directly to the CNS
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 that
motor neurons need to survive. That’s where SPINRAZA can help.
Watch Emma, a 7-year-old with SMA, explain how SPINRAZA works.