Individuals with SMA have a mutated survival motor neuron 1 (SMN1) gene and rely on the SMN2 gene to produce functional SMN protein. But the SMN2 gene can only produce about 10% of the full-length, functional protein that motor neurons need.
As an antisense oligonucleotide (ASO), SPINRAZA targets an underlying cause of motor neuron loss by binding to a specific sequence in the SMN2 gene to increase the production of functional SMN protein in the central nervous system (CNS).3,4
Delivery of SPINRAZA to the CNS helps infuse the motor neurons with nusinersen and maintain a high concentration and long half life in the spinal cord, where it's needed most.3,5,6
mRNA=messenger ribonucleic acid.
ASOs like SPINRAZA bind to specific nucleotide sequences. SPINRAZA binds to a sequence in the intron downstream of exon 7 of the SMN2 transcript3