Individual results may vary based on several factors, including severity of disease, initiation of treatment, and duration of therapy.


SMA is a disease of the CNS. SPINRAZA is delivered directly to the CNS.

Spinal muscular atrophy (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. 

Watch Emma, a 7-year-old with SMA, explain how SPINRAZA works.

SPINRAZA has been studied in the longest clinical trial program in SMA to date.