SMA is a disease of the central nervous system (CNS). SPINRAZA is delivered directly to the CNS1-3

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.1,3

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 CNS.3,4

Delivery of SPINRAZA to the CNS helps infuse the motor neurons with nusinersen and maintain an adequate concentration and long half-life in the spinal cord, where it's needed most3,5,6

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

Watch how SPINRAZA works to enhance the production of full-length SMN protein3

Download the patient materials to talk about SPINRAZA today.

Pediatric Brochure

for Adults

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