SMA is a disease of the CNS. SPINRAZA
is delivered directly to the CNS.1-3

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

SPINRAZA is an ASO that binds to a specific nucleotide sequence of exon 7 in the SMN2 transcript

ASOs like SPINRAZA bind to specific nucleotide sequences. SPINRAZA binds to a sequence in the intron downstream of exon 7 of the SMN2 transcript.3

Watch how SPINRAZA works to enhance the production of full-length SMN protein.3

Download the patient materials to talk about SPINRAZA today.

pediatric brochure

adult brochure

Did you know SPINRAZA is indicated
for both
pediatric and adult
patients with SMA?3