SPINRAZA increases survival motor neuron (SMN) protein, addressing the underlying cause of motor neuron loss in spinal muscular atrophy (SMA)
Wondering how SPINRAZA works?
Let Emma explain.
Individual with SMA
Missing or mutated
A “backup” to SMN1
In individuals with SMA, a mutated or deleted survival motor neuron 1 (SMN1) gene does not produce enough SMN protein to maintain the survival of motor neurons. These neurons control muscle activity by sending signals from the central nervous system (CNS).
Without a proper level of SMN protein, motor neurons in the spinal cord will be lost, preventing the body’s muscles from receiving signals from the brain. This can lead to progressive muscle weakness and wasting (atrophy).
Survival motor neuron 2 (SMN2) is a closely related “backup” gene that is not believed to be affected in SMA, but does not produce enough full length SMN protein on its own.
Individual taking SPINRAZA
Increased SMN protein production
BY TARGETING THE SMN2 GENE, SPINRAZA HAS BEEN SHOWN TO INCREASE ITS PRODUCTION OF SMN PROTEIN